Expansions of DNA repeats are very hard to analyze. A method developed by researchers at the Max Planck Institute for Molecular Genetics in Berlin allows for a detailed look at these previously inaccessible regions of the genome. It combines nanopore sequencing, stem cell, and CRISPR-Cas technologies. The method could improve the diagnosis of various congenital diseases and cancers in the future.

Large parts of the genome consist of monotonous regions where short sections of the genome repeat hundreds or thousands of times. But expansions of these "DNA repeats" in the wrong places can have dramatic consequences, like in patients with Fragile X syndrome, one of the most commonly identifiable hereditary causes of cognitive disability in humans. However, these repetitive regions are still regarded as an unknown territory that cannot be examined appropriately, even with modern methods.

A research team led by Franz-Josef Mller at the Max Planck Institute for Molecular Genetics in Berlin and the University Hospital of Schleswig-Holstein in Kiel recently shed light on this inaccessible region of the genome. Mller's team was the first to successfully determine the length of genomic tandem repeats in patient-derived stem cell cultures. The researchers additionally obtained data on the epigenetic state of the repeats by scanning individual DNA molecules. The method, which is based on nanopore sequencing and CRISPR-Cas technologies, opens the door for research into repetitive genomic regions, and the rapid and accurate diagnosis of a range of diseases.A gene defect on the X chromosomeIn Fragile X syndrome, a repeat sequence has expanded in a gene called FMR1 on the X chromosome. "The cell recognizes the repetitive region and switches it off by attaching methyl groups to the DNA," says Mller. These small chemical changes have an epigenetic effect because they leave the underlying genetic information intact. "Unfortunately, the epigenetic marks spread over to the entire gene, which is then completely shut down," explains Mller. The gene is known to be essential for normal brain development. He states: "Without the FMR1 gene, we see severe delays in development leading to varying degrees of intellectual disability or autism."

Female individuals are, in most cases, less affected by the disease, since the repeat region is usually located on only one of the two X chromosomes. Since the unchanged second copy of the gene is not epigenetically altered, it is able to compensate for the genetic defect. In contrast, males have only one X chromosome and one copy of the affected gene and display the full range of clinical symptoms. The syndrome is one of about 30 diseases that are caused by expanding short tandem repeats.

First precise mapping of short tandem repeats

In this study, Mller and his team investigated the genome of stem cells that were derived from patient tissue. They were able to determine the length of the repeat regions and their epigenetic signature, a feat that had not been possible with conventional sequencing methods. The researchers also discovered that the length of the repetitive region could vary to a large degree, even among the cells of a single patient.

The researchers also tested their process with cells derived from patients that contained an expanded repeat in one of the two copies of the C9orf72 gene. This mutation leads to one of the most common monogenic causes of frontotemporal dementia and amyotrophic lateral sclerosis. "We were the first to map the entire epigenetics of extended and unchanged repeat regions in a single experiment," says Mller. Furthermore, the region of interest on the DNA molecule remained physically wholly unaltered. "We developed a unique method for the analysis of single molecules and for the darkest regions of our genome - that's what makes this so exciting for me."

Tiny pores scan single molecules

"Conventional methods are limited when it comes to highly repetitive DNA sequences. Not to mention the inability to simultaneously detect the epigenetic properties of repeats," says Bjrn Brndl, one of the first authors of the publication. That's why the scientists used Nanopore sequencing technology, which is capable of analyzing these regions. The DNA is fragmented, and each strand is threaded through one of a hundred tiny holes ("nanopores") on a silicon chip. At the same time, electrically charged particles flow through the pores and generate a current. When a DNA molecule moves through one of these pores, the current varies depending on the chemical properties of the DNA. These fluctuations of the electrical signal are enough for the computer to reconstruct the genetic sequence and the epigenetic chemical labels. This process takes place at each pore and, thus, each strand of DNA.

Genome editing tools and bioinformatics illuminate "dark matter"Conventional sequencing methods analyze the entire genome of a patient. Now, the scientists designed a process to look at specific regions selectively. Brndl used the CRISPR-Cas system to cut DNA segments from the genome that contained the repeat region. These segments went through a few intermediate processing steps and were then funneled into the pores on the sequencing chip.

"If we had not pre-sorted the molecules in this way, their signal would have been drowned in the noise of the rest of the genome," says bioinformatician Pay Giesselmann. He had to develop an algorithm specifically for the interpretation of the electrical signals generated by the repeats: "Most algorithms fail because they do not expect the regular patterns of repetitive sequences." While Giesselmann's program "STRique" does not determine the genetic sequence itself, it counts the number of sequence repetitions with high precision. The program is freely available on the internet.

Numerous potential applications in research and the clinic"With the CRISPR-Cas system and our algorithms, we can scrutinize any section of the genome - especially those regions that are particularly difficult to examine using conventional methods," says Mller, who is heading the project. "We created the tools that enable every researcher to explore the dark matter of the genome," says Mller. He sees great potential for basic research. "There is evidence that the repeats grow during the development of the nervous system, and we would like to take a closer look at this."

The physician also envisions numerous applications in clinical diagnostics. After all, repetitive regions are involved in the development of cancer, and the new method is relatively inexpensive and fast. Mller is determined to take the procedure to the next level: "We are very close to clinical application."

Reference: Giesselmann et al. 2019.Analysis of short tandem repeat expansions and their methylation state with nanopore sequencing. Nature Biotechnology.DOI: https://doi.org/10.1038/s41587-019-0293-x.

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New Device Permits a Closer Look at Previously Inaccessible Areas of the Genome - Technology Networks

Salmonella is acting up in Michigan, and it could be a model for whats happening in other states, according to a new Michigan State University study.

The study, appearing in Frontiers in Medicine, documents a substantial uptick in antibiotic resistant strains, and consequently, longer hospital stays as doctors work to treat the increasing virulent pathogens.

If you get a salmonella infection that is resistant to antibiotics today, you are more likely to be hospitalized longer, and it will take you longer to recover, said Shannon Manning, MSU Foundation professor in theDepartment of Microbiology and Molecular Genetics and senior author of the study. We need better detection methods at the clinical level to identify resistant pathogens earlier so we can treat them with the right drugs the first time.

Losing a day or more to misdiagnosis or improper treatment allows symptoms to get worse. Doctors might kill off a subpopulation of bacteria that are susceptible, but the ones that are resistant grow stronger, she added.

Salmonella is a diverse group of bacterial pathogens that causes foodborne infections. Infected patients often develop diarrhea, nausea, vomiting and abdominal pain, though some infections are more severe and can be life threatening.

When it comes to treatments, each strain reacts differently to the range of antibiotics available for prescription by doctors. So getting it right the first time is crucial.

Specifically in Michigan, doctors are seeing more strains that are resistant to ampicillin, a common antibiotic prescribed to treat salmonella. Multidrug resistance, or resistance to more than three classes of antibiotics, has also increased in Michigan and could further complicate patient treatment plans.

Were still uncertain as to why this is happening; it could be that these antibiotics have been overprescribed in human and veterinary medicine and that possessing genes for resistance has allowed these bacteria to grow and thrive in the presence of antibiotics, Manning said. Each state has its own antibiotic-resistance issues. Its important that the medical profession remains vigilant to ever-changing patterns of resistance in salmonella and other foodborne pathogens, rather than look for a blanket national solution.

Historically, salmonella has affected young children and the elderly, but now theres been a rise in adult cases, suggesting that the epidemiology of the infections has changed in Michigan.

Diving into individual strains of salmonella, the team of scientists found that patients with Typhimurium were more likely to have resistant infections as were patients infected during the fall, winter or spring months.

Another distinction was revealed between the strains affecting people living in rural and urban areas. Enteritis infections tend to be higher in rural areas. This may be attributed to rural residents exposure to farm animals or untreated sources of water.

Each states salmonella population has its own personality; so every states approach to identifying disease drivers and effective treatments should be modified to reflect these traits.

Our results show the importance of surveillance, monitoring resistance frequencies and identifying risk factors specific to each state and region, Manning said. The trends that are revealed can lead to new prevention strategies.

Additional MSU researchers contributing to the study include Sanjana Mukherjee, the lead author, Chase Anderson and Rebekah Mosci. Scientists from Wayne State, Sparrow Hospitals and the Michigan Department of Health and Human Services also contributed to this research.

(Note for media: Please include a link to the original paper in online coverage: https://www.frontiersin.org/articles/10.3389/fmed.2019.00250/full)

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The nature of salmonella is changing and it's meaner - MSUToday

Fifty-two faculty members and researchers at the University of California San Diego are among the worlds most influential in their fields. The Web of Science Group, an information and technology provider for the global scientific research community, compiled its2019 Highly Cited Researcherslist of more than 6,000 scientists from around the world whose studies were among the top 1% of most-cited publications in their field over the past 11 years.

The number of highly cited researchers from UC San Diego increased by 13% over last years number of forty-six. The listing covers 21 fields of study as well as a cross-field category for researchers who are widely cited across multiple fields. UC San Diego had researchers listed in 14 fields, with the most cited in cross-field (23), followed by molecular biology and genetics (5), clinical medicine (4) and social sciences (4).

UC San Diego has some of the most dedicated, brilliant and hard-working faculty and researchers in the world. Their inclusion on the list of highly cited researchers is a measure of their impact in their respective fields of study as they continue to advance the frontiers of knowledge, said Chancellor Pradeep K. Khosla.

Of particular note is Director for the Center of Microbiome Innovation Rob Knights inclusion in three separate areas of study (biology and biochemistry, environment and ecology, microbiology). Out of 6,216 highly cited researchers, only 11 were cited in three fields, making Knight part of a super elite 0.3% of those listed.

There were also 23 Nobel laureates on the list, one of whom, Roger Tsien, was a distinguished professor of both Pharmacology in the School of Medicine and of Chemistry and Biochemistry at UC San Diego until his death in 2016. He shared the Nobel Prize in Chemistry with two others in 2008 for discovering and developing green fluorescent protein.

David Pendlebury, Senior Citation Analyst at the Web of Science Groups Institute for Scientific Information said that the highly cited researchers create gains for society, innovation and knowledge that make the world healthier, richer, more sustainable and more secure.

It is especially encouraging to see not only the number of highly cited researchers at the university, but the broad range of fields in which they are cited. It really speaks to the fact that UC San Diego conducts groundbreaking research across a wide range of disciplines, said Vice Chancellor for Research Sandra A. Brown. I congratulate everyone on their excellent research and contributions.

The 52 UC San Diego faculty members named by Web of Science and the fields of study in which they were cited are:

Gregory Aarons,social sciences

Ludmil Alexandrov, molecular biology and genetics

David Brenner,cross-field

Kristin Cadenhead,psychiatry/psychology

Kelli Cain, social sciences

Shu Chien, cross-field

Don Cleveland,neuroscience and behavior

Seth Cohen,chemistry

Pieter Dorrestein,cross-field

Mark Ellisman, cross-field

Mark Estelle,plant and animal science

Michael Folger, cross-field

Anthony Gamst, cross-field

Christopher Glass,molecular biology and genetics

Uri Gneezy,economics and business

Antonio Gonzalez, microbiology

Kun-Liang Guan,molecular biology and genetics

Trey Ideker,cross-field

Michael Karin,molecular biology and genetics

Arthur Kavanaugh,clinical medicine

Dusan Keres, space science

Rob Knight,(listed in 3 fields) biology and biochemistry, environment and ecology, microbiology

Razelle Kurzrock, clinical medicine

Lisa Levin, cross-field

Irene Litvan, neuroscience and behavior

Rohit Loomba, clinical medicine

Prashant Mali, biology and biochemistry

Eliezer Masliah, cross-field

Victor Nizet, cross-field

Jerrold Olefsky,cross-field

Bernhard Palsson,biology and biochemistry

Veerabhadran Ramanathan,cross-field

Bing Ren,molecular biology and genetics

Jeremy Rich, cross-field

Douglas Richman,cross-field

Michael Sailor,cross-field

James Sallis,social sciences

William Sandborn,clinical medicine

Bernd Schnabl, cross-field

Julian Schroeder,plant and animal science

Terrence Sejnowski, cross-field

Claude Sirlin, cross-field

Murray Stein,psychiatry/psychology

Steffanie Strathdee, cross-field

Roger Tsien, cross-field

Ming Tsuang,psychiatry/psychology

Joseph Wang,chemistry

Shang-Ping Xie,geosciences

Gene Yeo, cross-field

Kun Zhang, cross-field

Liangfang Zhang,cross-field

Yunde Zhao, plant and animal science

Shu-Hong Zhu, social sciences

You can read about Web of Sciences methodology on their website.

UC San Diegos Studio Ten 300 offers radio and television connections for media interviews with our faculty. For more information, email .(JavaScript must be enabled to view this email address).

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52 UC San Diego Researchers Are Most Highly Cited in Their Fields - UC San Diego Health

A thousand seemingly insignificant things change as an organism ages. Beyond the obvious signs like graying hair and memory problems are myriad shifts both subtler andmore consequential: Metabolic processes run less smoothly; neurons respond less swiftly; the replication of DNA grows faultier.

But while bodies mayseem to just gradually wear out, many researchers believe instead that aging is controlled at the cellular and biochemical level. They find evidence for this in the throngof biological mechanisms that are linked to aging but also conserved across species as distantly related as roundworms and humans. Whole subfields of research have grown up around biologists attempts to understand the relationships among the core genes involved in aging, which seem to connect highly disparate biological functions, like metabolism and perception. If scientists can pinpoint which of the changes in these processes induce aging, rather than result from it, it may be possible to intervene and extend the human life span.

So far, research has suggested that severely limiting calorie intake can have a beneficial effect, as can manipulating certain genes in laboratory animals. But recently in Nature, Bruce Yankner, a professor of genetics and neurology at Harvard Medical School, and his colleagues reported on a previously overlooked controller of life span: the activity level of neurons in the brain. In a series of experiments on roundworms, mice and human brain tissue, they found that a protein called REST, which controls the expression of many genes related to neural firing, also controls life span. They also showed that boosting the levels of the equivalent of REST in worms lengthens their lives by making their neurons fire more quietly and with more control. How exactly overexcitation of neurons might shorten life span remains to be seen, but the effect is real and its discovery suggests new avenues for understanding the aging process.

In the early days of the molecular study of aging, many people were skeptical that it was even worth looking into. Cynthia Kenyon, a pioneering researcher in this area at the University of California, San Francisco, has described attitudes in the late 1980s: The ageing field at the time was considered a backwater by many molecular biologists, and the students were not interested, or were even repelled by the idea. Many of my faculty colleagues felt the same way. One told me that I would fall off the edge of the Earth if I studied ageing.

That was because many scientists thought that aging (more specifically, growing old) must be a fairly boring, passive process at the molecular level nothing more than the natural result of things wearing out. Evolutionary biologists argued that aging could not be regulated by any complex or evolved mechanism because it occurs after the age of reproduction, when natural selection no longer has a chance to act. However, Kenyon and a handful of colleagues thought that if the processes involved in aging were connected to processes that acted earlier in an organisms lifetime, the real story might be more interesting than people realized. Through careful, often poorly funded work on Caenorhabditis elegans, the laboratory roundworm, they laid the groundwork for what is now a bustling field.

A key early finding was that the inactivation of a gene called daf-2 was fundamental to extending the life span of the worms. daf-2 mutants were the most amazing things I had ever seen. They were active and healthy and they lived more than twice as long as normal, Kenyon wrote in a reflection on these experiments. It seemed magical but also a little creepy: they should have been dead, but there they were, moving around.

This gene and a second one called daf-16 are both involved in producing these effects in worms. And as scientists came to understand the genes activities, it became increasingly clear that aging is not separate from the processes that control an organisms development before the age of sexual maturity; it makes use of the same biochemical machinery. These genes are important in early life, helping the worms to resist stressful conditions during their youth. As the worms age, modulation of daf-2 and daf-16 then influences their health and longevity.

These startling results helped draw attention to the field, and over the next two decades many other discoveries illuminated a mysterious network of signal transduction pathways where one protein binds another protein, which activates another, which switches off another and so on that, if disturbed, can fundamentally alter life span. By 1997, researchers had discovered that in worms daf-2 is part of a family of receptors that send signals triggered by insulin, the hormone that controls blood sugar, and the structurally similar hormone IGF-1, insulin-like growth factor 1; daf-16 was farther down that same chain. Tracing the equivalent pathway in mammals, scientists found that it led to a protein called FoxO, which binds to the DNA in the nucleus, turning a shadowy army of genes on and off.

That it all comes down to the regulation of genes is perhaps not surprising, but it suggests that the processes that control aging and life span are vastly complex, acting on many systems at once in ways that may be hard to pick apart. But sometimes, its possible to shine a little light on whats happening, as in the Yankner groups new paper.

Figuring out which genes are turned on and off in aging brains has long been one of Yankners interests. About 15 years ago, in a paper published in Nature, he and his colleagues looked at gene expression data from donated human brains to see how it changes over a lifetime. Some years later, they realized that many of the changes theyd seen were caused by a protein called REST. REST, which turns genes off, was mainly known for its role in the development of the fetal brain: It represses neuronal genes until the young brain is ready for them to be expressed.

But thats not the only time its active. We discovered in 2014 that [the REST gene] is actually reactivated in the aging brain, Yankner said.

To understand how the REST protein does its job, imagine that the network of neurons in the brain is engaged in something like the party game Telephone. Each neuron is covered with proteins and molecular channels that enable it to fire and pass messages. When one neuron fires, it releases a flood of neurotransmitters that excite or inhibit the firing of the next neuron down the line. REST inhibits the production of some of the proteins and channels involved in this process, reining in the excitation.

In their new study, Yankner and his colleagues report that the brains of long-lived humans have unusually low levels of proteins involved in excitation, at least in comparison with the brains of people who died much younger. This finding suggests that the exceptionally old people probably had less neural firing. To investigate this association in more detail, Yankners team turned to C. elegans. They compared neural activity in the splendidly long-lived daf-2 mutants with that of normal worms and saw that firing levels in the daf-2 animals were indeed very different.

They were almost silent. They had very low neural activity compared to normal worms, Yankner said, noting that neural activity usually increases with age in worms. This was very interesting, and sort of parallels the gene expression pattern we saw in the extremely old humans.

When the researchers gave normal roundworms drugs that suppressed excitation, it extended their life spans. Genetic manipulation that suppressed inhibition the process that keeps neurons from firing did the reverse. Several other experiments using different methods confirmed their results. The firing itself was somehow controlling life span and in this case, less firing meant more longevity.

Because REST was plentiful in the brains of long-lived people, the researchers wondered if lab animals without REST would have more neural firing and shorter lives. Sure enough, they found that the brains of elderly mice in which the Rest gene had been knocked out were a mess of overexcited neurons, with a tendency toward bursts of activity resembling seizures. Worms with boosted levels of their version of REST (proteins named SPR-3 and SPR-4) had more controlled neural activity and lived longer. But daf-2 mutant worms deprived of REST were stripped of their longevity.

It suggests that there is a conserved mechanism from worms to [humans], Yankner said. You have this master transcription factor that keeps the brain at what we call a homeostatic or equilibrium level it doesnt let it get too excitable and that prolongs life span. When that gets out of whack, its deleterious physiologically.

Whats more, Yankner and his colleagues found that in worms the life extension effect depended on a very familiar bit of DNA: daf-16. This meant that RESTs trail had led the researchers back to that highly important aging pathway, as well as the insulin/IGF-1 system. That really puts the REST transcription factor somehow squarely into this insulin signaling cascade, said Thomas Flatt, an evolutionary biologist at the University of Fribourg who studies aging and the immune system. REST appears to be yet another way of feeding the basic molecular activities of the body into the metabolic pathway.

Neural activity has been implicated in life span before, notes Joy Alcedo, a molecular geneticist at Wayne State University who studies the connections between sensory neurons, aging and developmental processes. Previous studies have found that manipulating the activity of even single neurons in C. elegans can extend or shorten life span. Its not yet clear why, but one possibility is that the way the worms respond biochemically to their environment may somehow trip a switch in their hormonal signaling that affects how long they live.

The new study, however, suggests something broader: that overactivity in general is unhealthy. Neuronal overactivity may not feel like anything in particular from the viewpoint of the worm, mouse or human, unless it gets bad enough to provoke seizures. But perhaps over time it may damage neurons.

The new work also ties into the idea that aging may fundamentally involve a loss of biological stability, Flatt said. A lot of things in aging and life span somehow have to do with homeostasis. Things are being maintained in a proper balance, if you will. Theres a growing consensus in aging research that what we perceive as the body slowing down may in fact be a failure to preserve various equilibria. Flatt has found that aging flies show higher levels of immune-related molecules, and that this rise contributes to their deaths. Keeping the levels in check, closer to what they might have been when the flies were younger, extends their lives.

The results may help explain the observation that some drugs used for epilepsy extend life span in lab animals, said Nektarios Tavernarakis, a molecular biologist at the University of Crete who wrote a commentary that accompanied Yankners recent paper. If overexcitation shortens life span, then medicines that systematically reduce excitation could have the opposite effect. This new study provides a mechanism, he said.

In 2014, Yankners laboratory also reported that patients with neurodegenerative diseases like Alzheimers have lower levels of REST. The early stages of Alzheimers, Yankner notes, involve an increase in neural firing in the hippocampus, a part of the brain that deals with memory. He and his colleagues wonder whether the lack of REST contributes to the development of these diseases; they are now searching for potential drugs that boost REST levels to test in lab organisms and eventually patients.

In the meantime, however, its not clear that people can do anything to put the new findings about REST to work in extending their longevity. According to Yankner, REST levels in the brain havent been tied to any particular moods or states of intellectual activity. It would be a misconception, he explained by email, to correlate amount of thinking with life span. And while he notes that there is evidence that meditation and yoga can have a variety of beneficial effects for mental and physical health, no studies show that they have any bearing on REST levels.

Why exactly do overexcited neurons lead to death? Thats still a mystery. The answer probably lies somewhere downstream of the DAF-16 protein and FoxO, in the genes they turn on and off. They may be increasing the organisms ability to deal with stress, reworking its energy production to be more efficient, shifting its metabolism into another gear, or performing any number of other changes that together add up a sturdier and longer-lived organism. It is intriguing that something as transient as the activity state of a neural circuit could have such a major physiological influence on something as protean as life span, Yankner said.

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Longevity Linked to Proteins That Calm Overexcited Neurons - Quanta Magazine

NEW YORK--(BUSINESS WIRE)--IVERIC bio, Inc. (NASDAQ: ISEE) announced today the appointment of Guangping Gao, PhD, as Chief Strategist, Gene Therapy. Dr. Gao brings over 30 years of scientific research experience in gene-based treatments. As one of the worlds leading gene therapy experts, Dr. Gaos highly distinguished career includes major contributions to the development of adeno-associated virus (AAV) gene delivery technology. Dr. Gao is the current President of the American Society of Gene and Cell Therapy (ASGCT). In his advisory role, Dr. Gao will help shape IVERIC bios gene therapy strategy going forward.

On behalf of the Board of Directors and management team at IVERIC bio, we are extremely excited to welcome Dr. Gao to IVERIC bio, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. Our goal at IVERIC bio is to become a leading retina company with a diversified portfolio addressing significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies. Dr. Gaos extensive background in gene therapy will be instrumental in shaping the direction of our gene therapy portfolio. It is a true privilege to work with him closely and benefit from his extensive expertise during these exciting times at our company.

I am delighted to have the opportunity to be a part of the IVERIC bio team, stated Dr. Gao. I am impressed with the progress that IVERIC bio had made with their diverse pipeline of novel gene therapy solutions to treat orphan inherited retinal diseases (IRDs). I look forward to working closely with the team to help build and maximize the potential of the Companys gene therapy programs.

Dr. Gao is a world-renowned scientist and researcher who is a pioneer in AAV gene delivery technology, stated Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio. We believe that the combination of Dr. Gaos expertise in gene therapy and our knowledge and experience in drug development for retinal diseases will help set the stage for the proficient execution of our gene therapy strategy, advancing it to the next stage of evolution. We will also continue to move forward our Zimura, complement C5 inhibitor, programs as expeditiously as possible.

About Guangping Gao, PhD

Dr. Gao is the Co-Director, Li Weibo Institute for Rare Diseases Research, Director, Horae Gene Therapy Center and Viral Vector Core, Professor of Microbiology and Physiological Systems and Penelope Booth Rockwell Professor in Biomedical Research, at the University of Massachusetts Medical School; an elected fellow of both the US National Academy of Inventors (NAI) and the American Academy of Microbiology; and the current President of the American Society of Gene and Cell Therapy.

Dr. Gao is an internationally recognized gene therapy researcher who has played a key role in the discovery and characterization of a new family of adeno-associated virus (AAV) serotypes, which was instrumental in reviving the gene therapy field, providing technology to enable potential treatments for many currently untreatable human diseases. For nearly 30 years of his scientific research career, Dr. Gao has primarily focused on molecular genetics and viral vector gene therapy for rare genetic diseases, with research encompassing disease gene cloning, causative mutation identification, pathomechanism investigation, animal modeling, novel viral vector discovery and engineering for in vivo gene delivery, vector biology, preclinical and clinical gene therapy product development, and viral vector manufacturing for preclinical and clinical gene therapy applications, as well as development of technology platforms for novel approaches for human gene therapy.

Dr. Gao has published 267 research papers, 6 book chapters, and 5 edited books and serves as Editor of Human Gene Therapy, Senior Editor of the Gene and Cell Therapy book series, Associate Editor of Signal Transduction and Targeted Therapy, and on the editorial boards of several other gene therapy and virology journals. Dr. Gao holds 135 patents with 239 additional patent applications pending. Dr. Gaos inventions have been licensed to and are currently in development by over ten pharmaceutical companies. Recently, Dr. Gao was ranked as #4 on Nature Biotechnologys list of the Worlds Top 20 Translational Researchers for 2017.

About IVERIC bio

IVERIC bio is a biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. Vision is Our Mission. For more information on the Company please visit http://www.ivericbio.com.

IVERIC bio Forward-looking Statements

Any statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about the implementation of its strategic plan to seek to address significant unmet medical needs in large market age-related retinal indications and inherited retinal diseases by utilizing both therapeutics and gene therapies, the timing, progress and results of clinical trials and other research and development activities, including manufacturing activities, the potential utility of its product candidates and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the initiation and the conduct and design of research programs and clinical trials, establishment of manufacturing capabilities, availability of data from these programs, reliance on university collaborators and other third parties, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.

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IVERIC bio Appoints Guangping Gao, PhD, Internationally Recognized Gene Therapy Pioneer as Chief Strategist, Gene Therapy - Business Wire

It is important to understand that the Molecular Therapy family of journals offers unprecedented opportunities to publish high-quality articles in gene and cell therapy, Herzog says.

MILWAUKEE (PRWEB) November 25, 2019

The American Society of Gene & Cell Therapy (ASGCT) announced today the appointment of Roland W. Herzog, Ph.D. and Gerhard Bauer as editors-in-chief of the Societys field-leading journals, Molecular Therapy and Molecular TherapyMethods & Clinical Development, respectively. Both editors will begin their positions effective January 1, 2020.

Herzog, currently the editor-in-chief of Molecular TherapyMethods & Clinical Development, will assume the same role for a five-year term at Molecular Therapy from Seppo Yl-Herttuala, M.D., Ph.D. at the conclusion of Yl-Herttualas term. Herzog is also a professor of pediatrics, Riley Children's Foundation Professor of Immunology, and director of the gene and cell therapy program at Indiana University.

It is important to understand that the Molecular Therapy family of journals offers unprecedented opportunities to publish high-quality articles in gene and cell therapy, Herzog says. My vision has continued and even further strengthened close coordination and collaboration between the journal editors and partnership with Cell Press and ASGCT.

Bauer replaces Herzog at the helm of Molecular TherapyMethods & Clinical Development after serving as an associate editor of the same journal for more than four years. Bauer is an assistant professor of hematology and oncology and director of the Good Manufacturing Practice (GMP) laboratory at the UC Davis Institute for Regenerative Cures.

Over the years that I have been looking at manuscripts submitted to the journal, I have always loved the articles dealing with translational research, getting the products ready for clinical applications, improving their utility and solving problems in the manufacturing of these products, Bauer says. Now, at a time where we have clinical efficacy and also commercially approved cell and gene therapy products that save lives, it is even more important to strengthen the translational research field.

ASGCTs official journal, Molecular Therapy is the leading journal for research in the areas of gene transfer, vector development and design, stem cell manipulation, development of gene-, peptide-, protein-, oligonucleotide-, and cell-based therapeutics to correct genetic and acquired diseases, vaccine development, pre-clinical target validation, safety/efficacy studies, and clinical trials. Molecular Therapy is dedicated to promoting the sciences in genetics, medicine, and biotechnology. Publishing important peer-reviewed research and cutting-edge reviews and commentaries, the journal continues to attract the best material in the field. Molecular Therapy's 2018 impact factor is 8.402.

The aim of Molecular TherapyMethods & Clinical Development is to build upon the success of Molecular Therapy in publishing important peer-reviewed methods and procedures, as well as translational advances in the broad array of fields under the molecular therapy umbrella. Molecular TherapyMethods & Clinical Development's 2018 impact factor is 4.875.

In addition to Molecular Therapy and Molecular Therapy Methods & Clinical Development, ASGCT also owns and operates two other sibling journals, Molecular TherapyNucleic Acids (2018 impact factor 5.919) and Molecular TherapyOncolytics (2018 impact factor 5.710).

About ASGCTThe American Society of Gene & Cell Therapy is the primary professional membership organization for scientists, physicians, patient advocates, and other professionals with interest in gene and cell therapy. Our members work in a wide range of settings including universities, hospitals, government agencies, foundations, biotechnology and pharmaceutical companies. ASGCT advances knowledge, awareness, and education leading to the discovery and clinical application of gene and cell therapies to alleviate human disease to benefit patients and society.

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Molecular Therapy Family of Journals Welcomes Roland W. Herzog and Gerhard Bauer as Editors-in-Chief - PR Web

One wanted to be a professional basketball player. Another wanted to be a dancer. And someone else wanted to be an astronaut. They all ended up making impacts in different careers.

For our series on Visionaries, The Times interviewed 12 people who are taking risks and working to change the world.

Technology visionary: Jeri Ellsworth

Ms. Ellsworth began playing pinball when she worked in a bowling alley as a teenager, and a manager there would occasionally give her a few free credits. Today, she has a collection of more than 70 pinball machines, but her passion has moved from the mechanical into a new digital augmented reality, which she believes will be the future of entertainment.

Ms. Ellsworth, 45, is a self-taught computer hacker and chip designer who recently started a new augmented reality gaming company, Tilt Five, based in San Jose, Calif. She is emblematic of a generation of Silicon Valley hobbyists who were passionate about computers and only later turned their passions into commercial enterprises. She originally gained visibility as an independent computer chip designer living in a rural ramshackle farmhouse in Yamhill, Ore. Read more

FOOD VISIONARY: Steve Palmer

Mr. Palmer has opened dozens of restaurants over the course of his career. He knows how to coax the best flavors out of a piece of fish, how to light a restaurant so all the customers look good and how to make a couple celebrating an anniversary feel special.

But 18 years ago, he was so strung out on alcohol and cocaine that his boss at the restaurant where he worked gave him an ultimatum: Get treatment or get fired.

He chose rehab. Now, Mr. Palmer, 50, is the managing partner of Indigo Road Hospitality Group, which employs about a thousand people in 20 restaurants and bars that stretch across four Southern states and Washington. Read more

Style visionary: Iris van Herpen

When many people think of couture they think of the most traditional, time-intensive kind of fashion; of seamstresses and tailors in white coats bent over intricate swathes of material painstakingly sewing by hand the way they have since the days of Charles Frederick Worth and Christian Dior (and Marie Antoinette, for that matter).

Ms. van Herpen, however, a 35-year-old Dutch designer who founded her own company in 2007, has always thought of something different.

She has thought of the way the sewing needle an early tool might translate into the tools of tomorrow; might, for example, connect to the 3-D printer and the laser cutter. She has explored such themes as biopiracy and magnetic motion; has combined mylar and copper with tulle and organza. Her dresses often appear to have their own energy field and look as though they are terraforming the body. Read more

Education Visionary: Jeff Duncan-Andrade

Sometimes it is the child who hates school who cares most about fixing its failings.

Which is how you can think of Mr. Duncan-Andrade. The youngest of seven who was born in Los Angeles and whose family moved from to Sacramento to Oregon to Oakland, Calif., during his school years found his teachers uninspiring. He only went to school to play ball.

But sports, specifically travel basketball, opened his eyes to the gulf between toxic coaches and those who forged relationships. Today, as an education activist, teacher, education coach, San Francisco State University associate professor and scholar, he is driving a public conversation that upends traditional beliefs about reforming schools and achieving equity. Read more

Culture Visionary: Shirin Neshat

Ms. Neshat has had an unusual trajectory to art world prominence. The artist left her native Iran as a teenager in the 1970s, before the revolution and the fall of the shah, and ended up studying at the University of California, Berkeley. By her own account, she didnt end up making art seriously until her early 30s. But Ms. Neshat, 62, has become one of the most distinct practitioners in the media of photography, video and film.

Now New York-based and working with several assistants in a large Bushwick studio, Ms. Neshat makes art that touches on topics like exile, political revolution and Irans past and future. The biggest exhibition of her career, Shirin Neshat: I Will Greet the Sun Again, runs from Oct. 19 to Feb. 16 at The Broad in Los Angeles. Read more

science visionary: Edith Heard

Twice in her life, Professor Heard has transformed scientific understanding.

She studies epigenetics changes to genetic activity that can be passed down to daughter cells without affecting the underlying genetic code. Among bees, for example, a simple difference in food supply means that a larva becomes either a worker or a queen.

Professor Heards first major advance was showing that these kinds of epigenetic changes can be incredibly dynamic, particularly as an embryo forms and matures. In her second, she revealed important information about how parts of the genome fold in space, allowing some genes to be activated and others silenced.

In January, Professor Heard took over as head of the European Molecular Biology Laboratory, or E.M.B.L., a scientific collaboration among 27 European countries. She received her bachelors degree in genetics from Cambridge Universitys Emmanuel College and her doctorate in biochemistry from the Imperial Cancer Research Fund in London. She is also a professor at the Collge de France. Read more

architecture visionary: Vo Trong Nghia

As city dwellers seek to soften the expanding urban jungle around them, architects are working harder to incorporate greenery and natural materials. But nobody is doing it quite like Mr. Nghia.

His firm, Vo Trong Nghia Architects, based in Ho Chi Minh City, infuses its work with lushly planted walls, hanging vines, structure-piercing trees, weathered stones and sunken landscapes. It also incorporates traditional Vietnamese building techniques, like complex bamboo trusses, perforated blocks, cooling water systems, shaded terraces and thatched roofs. Mr. Nghias firm also is expanding into prefab housing, urban farms, green towers, parks and urban plans around Asia.

All these efforts are infused with a resolute vision: the creation of architecture that merges nature, local vernacular and through modern materials and methods contemporary design. Mr. Nghia sees such work as a way not only to refine the urban environment, but also to provide a sense of peace in the world. Read more

agriculture visionary: Thomas Njeru

Few livelihoods offer as many paths to failure as agriculture. Throughout history, farmers have been at the mercy of nature be it weather, pests or crop diseases even as the survival of people and livestock depended on their success.

Growing up on a farm on the slopes of Mount Kenya, Mr. Njeru witnessed firsthand the devastating impact such setbacks had on the lives of small landholders, including his own family. His mother lectured him to work hard in school so he could one day leave the farm and land a good job in the city. Mr. Njeru followed that advice obediently until he did not.

Today, he is a co-founder and the chief financial officer of Pula, a four-year-old microinsurance firm that serves 1.7 million smallholder farms of 0.6 acres or less in 10 African countries and India. Read more

infrastructure visionary: james ehrlich

For Mr. Ehrlich, farm-to-table is just a starting point for the future.

The New York native, who migrated to Silicon Valley decades ago, is the founder and president of ReGen Villages, which seeks to create self-reliant ecosystems globally.

Mr. Ehrlich is anything but a typical real estate developer. Instead, his company, which he started in 2016, derives from his concern for the environment and his love of farming communities. His idea is to establish a far-reaching plan to create new neighborhoods that will generate their own power through solar photovoltaic panels, biomass and biogas from material, food and animal waste, and geothermal sources, to name but a few. Read more

sports visionary: Rich Luker

The sports world is filled with people whose job is to sell tickets, advertisements, sponsorships, luxury boxes and all manner of game-day experiences. The more, the better. Any fan will do.

Dr. Luker has tried to get those managers, supervisors and executives to look for something more profound, something he calls lifetime value.

To Dr. Luker, the most concrete iteration of that concept is the idea that passionate fans would get a tattoo of their favorite teams logo to show that it is part of their core identity.

This is just one of the discoveries he has made over the past 25 years as the founder of Luker On Trends, a sports polling company that works with many professional sports leagues and teams. Read more

BUSINESS VISIONARY: Andrew Kassoy

Sometimes its better not to know the obstacles that exist when creating something new.

That was certainly true for Mr. Kassoy, who, along with Jay Coen Gilbert and Bart Houlahan, two friends and roommates from Stanford University, wanted to encourage companies to focus on their employees and the environment. In 2006, they left their jobs and created B Lab, which certifies companies that operate for social good, in addition to trying to make money. Think Fair Trade for food or LEED certification for environmentally sustainable buildings.

Certified companies referred to as B Corps are businesses with verified social and environmental performances, including factors such as sustainability, income inequality and the impact on local communities. They are, essentially, companies that use business as a force of good, rather than focusing solely on profit maximization. Read more

Climate Visionary: Narasimha Rao

When policymakers, financiers and scientists describe the world decades from now, in the throes of climatic changes that we now only model, they emphasize what might be lost. They discuss the threats to gross domestic product, the havoc wrought by natural disasters or the runaway greenhouse gas emissions released by emerging national economies.

To Mr. Rao, a professor at the Yale School of Forestry and Environmental Studies specializing in energy systems analysis, that is a false choice, one that sacrifices justice on the altar of economic growth. Read more

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A Teacher, an Artist, a Scientist and More. Theyre All Visionaries. - The New York Times