Category Archives: Genetic Engineering

IMAGE:This is Charles Gersbach, assistant professor of biomedical engineering at Duke University. view more

DURHAM, N.C. -- Duke researchers have developed a new method to precisely control when genes are turned on and active.

The new technology allows researchers to turn on specific gene promoters and enhancers -- pieces of the genome that control gene activity -- by chemically manipulating proteins that package DNA. This web of biomolecules that supports and controls gene activity is known as the epigenome.

The researchers say having the ability to steer the epigenome will help them explore the roles that particular promoters and enhancers play in cell fate or the risk for genetic disease and it could provide a new avenue for gene therapies and guiding stem cell differentiation.

The study appears online April 6 in Nature Biotechnology.

"The epigenome is everything associated with the genome other than the actual genetic sequence, and is just as important as our DNA in determining cell function in healthy and diseased conditions," said Charles Gersbach, assistant professor of biomedical engineering at Duke. "That becomes immediately obvious when you consider that we have over 200 cell types, and yet the DNA in each is virtually the same. The epigenome determines which genes each cell activates and to what degree."

This genetic puppetmaster consists of DNA packaging proteins called histones and a host of chemical modifications -- either to these histones or the DNA itself -- that help determine whether a gene is on or off.

But Gersbach's team didn't have to modify the genes themselves to gain some control.

"Next to every gene is a DNA sequence called a promoter that controls its activity," explained Gersbach. "But there's also many other pieces of the genome called enhancers that aren't next to any genes at all, and yet they play a critical role in influencing gene activity too."

Timothy Reddy, assistant professor of biostatistics and bioinformatics at Duke, has spent the better part of a decade mapping millions of these enhancers across the human genome. There has not, however, been a good way to find out exactly what each one does. An enhancer might affect a gene next door or several genes across the genome -- or maybe none at all.

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Pulling the strings of our genetic puppetmasters

Bayers Marijn Dekkers (Christof Koepsel/Getty Images)

TOP STORIES

Endure if you must The Washington Posts takeout on tech gurus and venture capitalists with too much time on their hands trying to extend life (though most of the possible stuff they talk about are simply medical treatments not invented). Accompanying the story is a somewhat interesting game The Post created in which you drag stem cells into your brain and so on to extend your life.

I know its a week away but you should probably start watching the HIMSS 2015 hashtag now.

LIFE SCIENCE

A long but worthwhile read on a moratorium and proper path toward human genetic engineering.

In the long run, I believe the permissibility of using germline genomic modification to make babies will be, and should be, a political issue. Right now, I suspect I would opt for regulating it on a safety/benefit basis, allowing it only when the potential benefits outweighed the risks. But I might change my mind, either because of newly discovered facts or well-made arguments. Importantly, though I do not think that my view should govern. The people, through their governments, should govern.

Medtronic has invested $2 million in DreaMed Diabetes and will be using its artificial pancreas technology in is insulin pumps.

I hope you didnt miss The Wall Street Journals look at Bayer and its continued focus on its health and agriculture businesses. Bayer is dumping its $10 billion specialty plastics business.

Still, some analysts are skeptical that Bayers drug pipeline is strong enough to deliver many new products with selling power like the current wave. But Bayer expects at least three new drugs in midstage clinical testing, including two for chronic heart failure, to advance this year. Strong data is expected for those trials, said Ali Al-Bazergan, an analyst at Datamonitor Healthcare in London.

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Going deep on life extension investments and human genetic engineering (Morning Read)

(New York - March 25, 2015) Induced pluripotent stem cells (iPSCs) -- adult cells reprogrammed back to an embryonic stem cell-like state--may better model the genetic contributions to each patient's particular disease. In a process called cellular reprogramming, researchers at Icahn School of Medicine at Mount Sinai have taken mature blood cells from patients with myelodysplastic syndrome (MDS) and reprogrammed them back into iPSCs to study the genetic origins of this rare blood cancer. The results appear in an upcoming issue of Nature Biotechnology.

In MDS, genetic mutations in the bone marrow stem cell cause the number and quality of blood-forming cells to decline irreversibly, further impairing blood production. Patients with MDS can develop severe anemia and in some cases leukemia also known as AML. But which genetic mutations are the critical ones causing this disease?

In this study, researchers took cells from patients with blood cancer MDS and turned them into stem cells to study the deletions of human chromosome 7 often associated with this disease.

"With this approach, we were able to pinpoint a region on chromosome 7 that is critical and were able to identify candidate genes residing there that may cause this disease," said lead researcher Eirini Papapetrou, MD, PhD, Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai.

Chromosomal deletions are difficult to study with existing tools because they contain a large number of genes, making it hard to pinpoint the critical ones causing cancer. Chromosome 7 deletion is a characteristic cellular abnormality in MDS and is well-recognized for decades as a marker of unfavorable prognosis. However, the role of this deletion in the development of the disease remained unclear going into this study.

Understanding the role of specific chromosomal deletions in cancers requires determining if a deletion has observable consequences as well as identifying which specific genetic elements are critically lost. Researchers used cellular reprogramming and genome engineering to dissect the loss of chromosome 7. The methods used in this study for engineering deletions can enable studies of the consequences of alterations in genes in human cells.

"Genetic engineering of human stem cells has not been used for disease-associated genomic deletions," said Dr. Papapetrou. "This work sheds new light on how blood cancer develops and also provides a new approach that can be used to study chromosomal deletions associated with a variety of human cancers, neurological and developmental diseases."

Reprogramming MDS cells could provide a powerful tool to dissect the architecture and evolution of this disease and to link the genetic make-up of MDS cells to characteristics and traits of these cells. Further dissecting the MDS stem cells at the molecular level could provide insights into the origins and development of MDS and other blood cancers. Moreover, this work could provide a platform to test and discover new treatments for these diseases.

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This study was supported by grants from the National Institutes of Health, the American Society of Hematology, the Sidney Kimmel Foundation for Cancer Research, the Aplastic Anemia & MDS International Foundation, the Ellison Medical Foundation, the Damon Runyon Cancer Research Foundation, the University of Washington Royalty Research Fund, and a John H. Tietze Stem Cell Scientist Award.

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Researchers discover genetic origins of myelodysplastic syndrome using stem cells

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Newswise (New York March 25, 2015) Induced pluripotent stem cells (iPSCs)adult cells reprogrammed back to an embryonic stem cell-like statemay better model the genetic contributions to each patient's particular disease. In a process called cellular reprogramming, researchers at Icahn School of Medicine at Mount Sinai have taken mature blood cells from patients with myelodysplastic syndrome (MDS) and reprogrammed them back into iPSCs to study the genetic origins of this rare blood cancer. The results appear in an upcoming issue of Nature Biotechnology.

In MDS, genetic mutations in the bone marrow stem cell cause the number and quality of blood-forming cells to decline irreversibly, further impairing blood production. Patients with MDS can develop severe anemia and in some cases leukemia also known as AML. But which genetic mutations are the critical ones causing this disease?

In this study, researchers took cells from patients with blood cancer MDS and turned them into stem cells to study the deletions of human chromosome 7 often associated with this disease.

With this approach, we were able to pinpoint a region on chromosome 7 that is critical and were able to identify candidate genes residing there that may cause this disease, said lead researcher Eirini Papapetrou, MD, PhD, Department of Oncological Sciences, Icahn School of Medicine at Mount Sinai.

Chromosomal deletions are difficult to study with existing tools because they contain a large number of genes, making it hard to pinpoint the critical ones causing cancer. Chromosome 7 deletion is a characteristic cellular abnormality in MDS and is well-recognized for decades as a marker of unfavorable prognosis. However, the role of this deletion in the development of the disease remained unclear going into this study.

Understanding the role of specific chromosomal deletions in cancers requires determining if a deletion has observable consequences as well as identifying which specific genetic elements are critically lost. Researchers used cellular reprogramming and genome engineering to dissect the loss of chromosome 7. The methods used in this study for engineering deletions can enable studies of the consequences of alterations in genes in human cells.

Genetic engineering of human stem cells has not been used for disease-associated genomic deletions, said Dr. Papapetrou. This work sheds new light on how blood cancer develops and also provides a new approach that can be used to study chromosomal deletions associated with a variety of human cancers, neurological and developmental diseases.

Reprogramming MDS cells could provide a powerful tool to dissect the architecture and evolution of this disease and to link the genetic make-up of MDS cells to characteristics and traits of these cells. Further dissecting the MDS stem cells at the molecular level could provide insights into the origins and development of MDS and other blood cancers. Moreover, this work could provide a platform to test and discover new treatments for these diseases.

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Mount Sinai Researchers Discover Genetic Origins of Myelodysplastic Syndrome Using Stem Cells

Designer babies are on their way, said experts in genetic engineering as they called for a global ban on the practice.

It is thought that studies involving the use of genome-editing tools to modify the DNA of human embryos will be published shortly, said the authors of a paper in Nature.

The articles lead author, Professor Jennifer Doudna of the University of California at Berkeley, led the team that developed the gene-editing technique that she now wants restricted.

She and her colleagues have now warned of the ethical and safety implications of research that could lead to the birth of what laymen might term super humans.

In our view, genome editing in human embryos using current technologies could have unpredictable effects on future generations, they said. This makes it dangerous and ethically unacceptable. Such research could be exploited for non-therapeutic modifications.

DNA can be edited far more precisely than ever before using Crispr-Cas9 (Credit: Mehmet Pinarci/Sendercorp)

It is possible, for example, for the technology to make unintended changes to DNA, The New York Times reported.

But they are also worried that a public backlash could halt work on disease fighting techniques in somatic (non-reproductive) cells.

Genome-editing technologies may offer a powerful approach to treat many human diseases, including HIV/Aids, haemophilia, sickle-cell anaemia and several forms of cancer, they said.

Scientists at the Hubrecht Institute in the Netherlands reported in Cell Stem Cell two years ago that the technique could repair the cystic fibrosis mutation.

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'Ban DNA Editing Of Sperm And Eggs'

13 hours ago by Robert Sanders The bacterial enzyme Cas9 is the engine of RNA-programmed genome engineering in human cells. Credit: Jennifer Doudna/UC Berkeley

A group of 18 scientists and ethicists today warned that a revolutionary new tool to cut and splice DNA should be used cautiously when attempting to fix human genetic disease, and strongly discouraged any attempts at making changes to the human genome that could be passed on to offspring.

Among the authors of this warning is Jennifer Doudna, the co-inventor of the technology, called CRISPR-Cas9, which is driving a new interest in gene therapy, or "genome engineering." She and colleagues co-authored a perspective piece that appears in the March 20 issue of Science, based on discussions at a meeting that took place in Napa on Jan. 24. The same issue of Science features a collection of recent research papers, commentary and news articles on CRISPR and its implications.

"Given the speed with which the genome engineering field is evolving, our group concluded that there is an urgent need for open discussion of the merits and risks of human genome modification by a broad cohort of scientists, clinicians, social scientists, the general public and relevant public entities and interest groups," the authors wrote.

Doudna, director of UC Berkeley's Innovative Genomics Initiative, was joined by five current and two former UC Berkeley scientists, plus David Baltimore, a Nobel laureate and president emeritus of the California Institute of Technology, Stanford Nobelist Paul Berg and eminent scientists from UC San Francisco, Stanford, Harvard and the universities of Wisconsin and Utah. Several of these scientists are currently involved in gene therapy to cure inherited diseases.

Latest of many warnings

Such warnings have been issued numerous times since the dawn of genetic engineering in 1975, but until now the technology to actually fix genetic defects was hard to use.

"However, this limitation has been upended recently by the rapid development and widespread adoption of a simple, inexpensive and remarkably effective genome engineering method known as CRISPR-Cas9," the scientists wrote. "The simplicity of the CRISPR-Cas9 system enables any researcher with knowledge of molecular biology to modify genomes, making feasible many experiments that were previously difficult or impossible to conduct."

Correcting genetic defects

Scientists today are changing DNA sequences to correct genetic defects in animals as well as cultured tissues generated from stem cells, strategies that could eventually be used to treat human disease. The technology can also be used to engineer animals with genetic diseases mimicking human disease, which could lead to new insights into previously enigmatic disorders.

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Scientists call for caution in using DNA-editing technology

BERKELEY

A group of 18 scientists and ethicists today warned that a revolutionary new tool to cut and splice DNA should be used cautiously when attempting to fix human genetic disease, and strongly discouraged any attempts at making changes to the human genome that could be passed on to offspring.

Among the authors of this warning is Jennifer Doudna, the co-inventor of the technology, called CRISPR-Cas9, which is driving a new interest in gene therapy, or genome engineering. She and colleagues co-authored a perspective piece that appears in the March 20 issue of Science, based on discussions at a meeting that took place in Napa on Jan. 24. The same issue ofSciencefeatures a collection of recent research papers, commentary and news articles on CRISPR and its implications.

Given the speed with which the genome engineering field is evolving, our group concluded that there is an urgent need for open discussion of the merits and risks of human genome modification by a broad cohort of scientists, clinicians, social scientists, the general public and relevant public entities and interest groups, the authors wrote.

Doudna, director of UC Berkeleys Innovative Genomics Initiative, was joined by five current and two former UC Berkeley scientists, plus David Baltimore, a Nobel laureate and president emeritus of the California Institute of Technology, Stanford Nobelist Paul Berg and eminent scientists from UC San Francisco, Stanford, Harvard and the universities of Wisconsin and Utah. Several of these scientists are currently involved in gene therapy to cure inherited diseases.

Such warnings have been issued numerous times since the dawn of genetic engineering in 1975, but until now the technology to actually fix genetic defects was hard to use.

However, this limitation has been upended recently by the rapid development and widespread adoption of a simple, inexpensive and remarkably effective genome engineering method known as CRISPR-Cas9, the scientists wrote. The simplicity of the CRISPR-Cas9 system enables any researcher with knowledge of molecular biology to modify genomes, making feasible many experiments that were previously difficult or impossible to conduct.

Correcting genetic defects

Scientists today are changing DNA sequences to correct genetic defects in animals as well as cultured tissues generated from stem cells, strategies that could eventually be used to treat human disease. The technology can also be used to engineer animals with genetic diseases mimicking human disease, which could lead to new insights into previously enigmatic disorders.

The CRISPR-Cas9 tool is still being refined to ensure that genetic changes are precisely targeted, Doudna said. Nevertheless, the authors met to initiate an informed discussion of the uses of genome engineering technology, and to identify proactively those areas where current action is essential to prepare for future developments. We recommend taking immediate steps toward ensuring that the application of genome engineering technology is performed safely and ethically.

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Scientists urge caution in using new CRISPR technology to treat human genetic disease

IMAGE:BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD.... view more

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 26, 2015--The promise for using mesenchymal stem cells (MSC) to repair cartilage damage caused by osteoarthritis depends on the MSC being able to attach efficiently to the defective cartilage. A novel laboratory model in which artificially created cartilage lesions and labeled MSC were used to test factors that might improve MSC binding and the effectiveness of future MSC-based therapies is described in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the BioResearch Open Access website.

In the article "1 Integrins Mediate Attachment of Mesenchymal Stem Cells to Cartilage Lesions," D. Zwolanek, PhD, and coauthors, University of Veterinary Medicine (Vienna, Austria), University of Cologne Medical Faculty (Germany), University Medical Center Rotterdam (The Netherlands) present the results of experiments using a combination of ex vivo and in vivo model systems of defective cartilage. They studied the effects of serum, plasma hyaluronic acid, and various cell adhesion-related proteins such as integrins on the attachment of MSC to the extracellular matrix of the cartilage.

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal led by Editor-in-Chief Robert Lanza, MD, Chief Scientific Officer, Advanced Cell Technology, Inc. and Editor Jane Taylor, PhD. The Journal provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMed Central. All journal content is available on the BioResearch Open Access website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many areas of science and biomedical research, including DNA and Cell Biology, Tissue Engineering, Stem Cells and Development, Human Gene Therapy, HGT Methods, and HGT Clinical Development, and AIDS Research and Human Retroviruses. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Integrins are essential in stem cell binding to defective cartilage for joint regeneration

IMAGE:AIDS Research and Human Retroviruses, published monthly in print and online, presents papers, reviews, and case studies documenting the latest developments and research advances in the molecular biology of HIV... view more

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 6, 2015--Timothy Ray Brown, long known only as the "Berlin Patient" had HIV for 12 years before he became the first person in the world to be cured of the infection following a stem cell transplant in 2007. He recalls his many years of illness, a series of difficult decisions, and his long road to recovery in the first-person account, "I Am the Berlin Patient: A Personal Reflection," published in AIDS Research and Human Retroviruses, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is part of a special issue on HIV Cure Research and is available free on the AIDS Research and Human Retroviruses website.

Brown's Commentary describes the bold experiment of using a stem cell donor who was naturally resistant to HIV infection to treat the acute myeloid leukemia (AML) diagnosed 10 years after he became HIV-positive. The stem cell donor had a specific genetic mutation called CCR5 Delta 32 that can protect a person against HIV infection. The virus is not able to enter its target, the CD4 cells. After the stem cell transplant, Brown was able to stop all antiretroviral treatment and the HIV has not returned.

"This is the first time that we get to read this important story written by the man who lived it," says Thomas Hope, PhD, Editor-in-Chief of AIDS Research and Human Retroviruses and Professor of Cell and Molecular Biology at Northwestern University, Feinberg School of Medicine, Chicago, IL. "It is a unique opportunity to share in the human side of this transformative experience."

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About the Journal

AIDS Research and Human Retroviruses, published monthly in print and online, presents papers, reviews, and case studies documenting the latest developments and research advances in the molecular biology of HIV and SIV and innovative approaches to HIV vaccine and therapeutic drug research, including the development of antiretroviral agents and immune-restorative therapies. The content also explores the molecular and cellular basis of HIV pathogenesis and HIV/HTLV epidemiology. The Journal features rapid publication of emerging sequence information and reports on clinical trials of emerging HIV therapies. Tables of content and a sample issue may be viewed on the AIDS Research and Human Retroviruses website.

About the Publisher

Mary Ann Liebert, Inc., publishers/ is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including AIDS Patient Care and STDs, Viral Immunology, and Journal of Interferon & Cytokine Research. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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The 'Berlin patient,' first and only person cured of HIV, speaks out

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Michael Hadjiargyrou, Ph.D., of New York Institute of Technologys College of Arts and Sciences, an expert in engineering new musculoskeletal tissue, is available to discuss advanced bio-science predictions for 2015.

Undoubtedly, next year we will continue to see research advances in the realm of Tissue Engineering/Regenerative Medicine (TERM), says Hadjiargyrou, who specializes in molecular and cell biology, genetic engineering, biomaterials and stem cell research. Specifically, we will witness the formation of more tissues and possibly even organs fabricated in the laboratory with the use of 3D printers (Bioprinters).

Hadjiargyrou specifically identifies the heart valve, blood vessel, trachea, kidney, and liver, as the tissue or organs that will be printed with the use of 3D printers; kidneys, livers and hearts are most in demand.

Additionally, the successful transplantation of some of these laboratory tissues and organs will be achieved, particularly in Europe, as they have been more active in transplantation of biomaterials. With the emergence of such breakthroughs, we will begin to see more and more clinical and even cosmetic applications of TERM.

Hadjiargyrou focuses on studying the molecular mechanisms involved in bone regeneration as a way to better understand the healing of fractures. Hadjiargyrou teaches general biology, genetic engineering, contemporary biotechnology and biomedical research in Old Westbury, NY.

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NYIT Expert Predicts Growth in Demand for 3D Kidneys, Livers and Hearts