Category Archives: Genetic Engineering

John Sterling | 05/01/2014

The following article, reproduced in full below, was originally published at Genetic Engineering & Biotechnology News.

Its been a hot year for biotech! As G. Steven Burrill, CEO of Burrill & Co., noted in a recent report, life science firms raised $2.9 billion in new equity capital globally from public investors in February. This included $1.1 billion raised by 18 companies that completed initial public offerings and $1.8 billion raised by 23 companies that completed follow-on offerings during the month.

In the U.S., 16 life sciences companies raised $959 million through IPOs and 22 companies raised $1.75 billion through follow-on offerings on U.S. exchanges during February, making the month the biggest for IPOs in terms of the number of completed deals since February 2000!

Why the excitement? Promising new biotherapeutics are emerging from the drug pipeline. Advances in stem cell research and regenerative medicine are occurring at a rapid pace. And OMICS technologies (e.g., genomics, proteomics, metabolomics, transcriptomics, glycomics, and lipomics), originally developed and used in the lab, are now making their way into clinical medicine, truly ready to usher in an era of personalized medicine.

The 2014 BIO International Convention will be held in San Diego this June. As usual, the BIO conference committee did a superb job in putting together a first-class program that covers a wide range of topics with something to offer everyone involved in biotech R&D or commercialization. Its been a tough call this year but here are my picks for the top 10 cant miss sessions at the conference.

To learn more about the program and available registration packages for Convention, please visithere

John Sterling is editor-in-chief of Genetic Engineering & Biotechnology News (GEN).

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GENs Top 10 Session Picks for the 2014 BIO International Convention

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PUBLIC RELEASE DATE:

17-Apr-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, April 17, 2014The development of stem cell therapies to cure a variety of diseases depends on the ability to characterize stem cell populations based on cell surface markers. Researchers from the Finnish Red Cross have discovered a new marker that is highly expressed in a type of stem cells derived from human umbilical cord blood, which they describe in an article in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

Heli Suila and colleagues, Finnish Red Cross Blood Service, Helsinki, Finland present evidence to show that the glycan O-GLcNAc, is present on the surface of stem cells and is part of a stem cell-specific surface signature. In the article "Extracellular O-Linked N-Acetylglucosamine Is Enriched in Stem Cells Derived from Human Umbilical Cord Blood" the authors suggest that the glycan plays a crucial role in a cell signaling pathway that regulates embryonic development.

"This work is particularly interesting as epidermal growth factor domains are found on the Notch receptors, suggesting that these novel glycans may be involved in Notch receptor signaling pathways in stem cells," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland.

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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 PubMedCentral. All journal content is available on the BioResearch Open Access website.

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Novel marker discovered for stem cells derived from human umbilical cord blood

PUBLIC RELEASE DATE:

10-Apr-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, April 10, 2014In early fetal development, skin wounds undergo regeneration and healing without scar formation. This mechanism of wound healing later disappears, but by studying the fetal stem cells capable of this scarless wound healing, researchers may be able to apply these mechanisms to develop cell-based approaches able to minimize scarring in adult wounds, as described in a Critical Review article published in Advances in Wound Care, a monthly publication from Mary Ann Liebert, Inc., publishers and an Official Journal of the Wound Healing Society. The article is available free on the Advances in Wound Care website.

Michael Longaker, Peter Lorenz, and co-authors from Stanford University School of Medicine and John A. Burns School of Medicine, University of Hawaii, Honolulu, describe a new stem cell that has been identified in fetal skin and blood that may have a role in scarless wound healing. In the article "The Role of Stem Cells During Scarless Skin Wound Healing", the authors propose future directions for research to characterize the differences in wound healing mechanisms between fetal and adult skin-specific stem cells.

"This work comes from the pioneers in the field and delineates the opportunities towards scarless healing in adults," says Editor-in-Chief Chandan K. Sen, PhD, Professor of Surgery and Director of the Comprehensive Wound Center and the Center for Regenerative Medicine and Cell-Based Therapies at The Ohio State University Wexner Medical Center, Columbus, OH.

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About the Journal

Advances in Wound Care is a monthly journal published online and in print that reports the latest scientific discoveries, translational research, and clinical developments in acute and chronic wound care. Each issue provides a digest of the latest research findings, innovative wound care strategies, industry product pipeline, and developments in biomaterials and skin and tissue regeneration to optimize patient outcomes. The broad scope of applications covered includes limb salvage, chronic ulcers, burns, trauma, blast injuries, surgical repair, skin bioengineering, dressings, anti-scar strategies, diabetic ulcers, ostomy, bedsores, biofilms, and military wound care. Complete tables of content and a sample issue may be viewed on the Advances in Wound Care website.

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Scarless wound healing -- applying lessons learned from fetal stem cells

PUBLIC RELEASE DATE:

1-Apr-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, April 1, 2014 -- Rare, very small embryonic-like stem cells (VSELs) isolated from human adult tissues could provide a new source for developing regenerative therapies to repair complex tissues damaged by disease or trauma. The ability of these most-primitive, multipotent stem cells to differentiate into bone, neurons, connective tissue, and other cell types, and the proper criteria for identifying and isolating VSELs, are described in two articles in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available on the Stem Cells and Development website.

Russ Taichman and coauthors, University of Michigan (Ann Arbor) and NeoStem (New York, NY), implanted human VSELs into the cavity created by a cranial wound and provided the first demonstration that they could generate tissue structures containing multiple cell types. Their work is presented in "Human and Murine Very Small Embryonic-Like (VSEL) Cells Represent Multipotent Tissue Progenitors, In Vitro and In Vivo."

Malwina Suszynska et al., University of Louisville, KY, and Pomeranian Medical University (Szczecin) and Jagiellonian University (Krakow), Poland, explore the challenges in isolating these rare stem cells and the importance of not confusing VSELs with other types of embryonic or reprogrammed adult pluripotent stem cells, or with monopotent adult stem cells. In the Issues in Development article "The Proper Criteria for Identification and Sorting of Very Small Embryonic-Like Stem Cells (VSELs), and Some Nomenclature Issues," the authors present the most current descriptions and terminology for characterizing VSELs.

"I find the data presented by the Taichman group to be compelling and challenging. However, the current debate as to the significance of the body of publications concerning VSELs can only be resolved by a cooperative investigation across laboratories using identical methodologies and source materials," says Editor-in-Chief Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

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About the Journal

Stem Cells and Development is an authoritative peer-reviewed journal published 24 times per year in print and online. The Journal is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. Complete tables of content and a free sample issue may be viewed on the Stem Cells and Development website.

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First evidence that very small embryonic-like stem cells

PUBLIC RELEASE DATE:

25-Mar-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 24, 2014The curative and therapeutic potential of mesenchymal stem cells (MSCs) offers much promise, as these multipotent cells are currently being tested in more than 300 clinical trials in a range of diseases. A new, easier, and more reliable way to make large quantities of highly potent MSCs could accelerate progress toward their use in regenerative medicine, as described in an article in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Stem Cells and Development website.

Robert Lanza, MD and colleagues from Advanced Cell Technology (Marlborough, MA) and the David Geffen School of Medicine, UCLA (Los Angeles, CA), developed an innovative method for deriving MSCs from human embryonic stem cells (hESCs) through the use of a developmental precursor called the hemangioblast. They describe the technique and evidence of therapeutic efficacy using the hESC-MSCs to treat mouse models of lupus erythematosus and uveitis in the article "Mesenchymal Stem Cell Population Derived from Human Pluripotent Stem Cells Displays Potent Immunomodulatory and Therapeutic Properties."

"This new population of hESC-derived MSCs has a 30,000-fold greater proliferative capacity than bone marrow-derived MSCs," says Dr. Lanza, Chief Scientific Officer, Advanced Cell Technology. "In addition to being easy to derive in very large numbers, they are more youthful and live much longer." Dr. Lanza is Editor-in-Chief of BioResearch Open Access, a peer-reviewed open access journal from Mary Ann Liebert, Inc., publishers that provides a rapid-publication forum for a broad range of scientific topics.

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About the Journal

Stem Cells and Development is an authoritative peer-reviewed journal published 24 times per year online with Open Access options and in print. Led by Editor-in-Chief Graham C. Parker, PhD, the Journal is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. Complete tables of content and a sample issue may be viewed on the Stem Cells and Development website.

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New method yields potent, renewable human stem cells with promising therapeutic properties

PUBLIC RELEASE DATE:

24-Mar-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 24, 2014Marina Cavazzana, MD, PhD, Paris Descartes University, France and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, UK, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers, is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel* and publishing a Pioneer Perspective by the award recipients

Dr. Cavazzana has been at the forefront of advances in treating life-threatening inherited diseases of the immune system with gene therapy, using a patient's own modified stem cells. She describes the translation of this work to the clinic and its ongoing advances and novel applications in the article "Hematopoetic Stem Cell Gene Therapy: Progress on the Clinical Front." The article by Dr. Cavazzana is available free on the Human Gene Therapy website at http://online.liebertpub.com/doi/full/10.1089/hum.2014.2504.

A pioneer of gene therapy in the UK, Dr. Thrasher has been at the leading edge of basic science research on the function of therapeutic genes for inherited disorders and the development of viral vectors to deliver them to affected patients. He has collaborated on gene therapy clinical trials targeting immunodeficiency disorders with groups in Europe and the USA.

"Cell therapy and gene therapy are advancing together to improve patient care," says Dr. Cavazzana. "We can expect to be able to rebuild a new immune system not only in primary immunodeficiencies but also in severe acquired clinical conditions (such as those in HIV-1-infected patients)."

"I've seen some very exciting times in the field, from the first evidence that biochemical defects can be corrected in vitro, to some remarkable clinical successes in patients with devastating diseases. I look forward with huge enthusiasm to the exciting developments on the horizon, which are likely to impact on more patients with an even wider range of disorders," says Dr. Thrasher.

"These pioneers contributed to the first real clinical successes of gene therapy through their work in inherited immune deficiency disorders," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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Pioneer Award recipients Marina Cavazzana and Adrian Thrasher recognized for advancing gene therapy to the clinic for ...

What many didnt realise was that the freaky looking ear was never grown, had nothing to do with genetic engineering and wasnt really a scientific breakthrough at all! Instead, it served as the publics introduction to the new field of tissue engineering, through which researchers attempt to create replacement tissues in the laboratory by combining resorbable materials with stem cells.

Tissue engineers, like those in my laboratory at Kings College London, work to build everything from cartilage to fix creaky arthritic knees to coronary arteries to patch up heart patients. What looked like a human ear grown on a mouse was simply what we call a scaffold, an implantable 3D structure made of a plastic that safely dissolves in the body.

Twenty years later, a UCL-based team led by Dr Patrizia Ferretti is continuing to build on this work to reconstruct ears. Surgeons currently treat microtia, a condition in which children are born with a malformed or missing ear, by taking cartilage from the patients rib and implanting it in the head to form something that looks like an ear.

Dr Ferretti hopes to eliminate the need for this second cartilage-harvesting surgery by growing ear cartilage in the laboratory.

The difference here is that whereas in the 1990s tissue engineers thought that merely forming a scaffold of the correct shape and size would allow us to create a tissue, we now understand that a stem cells perception of its nano-environment plays an important role in determining the tissue it creates.

In short, we can now tailor a scaffold with nano-cues that tell a stem cell to become a liver cell instead of lung.

Dr Ferrettis scaffold does just this. Her team utilises a new nanocaged POSS-PCU scaffold to coax stem cells collected from fat to form cartilage whilst the scaffold slowly melts away.

This exciting material came to light in 2011 when it was used to replace the windpipe of a patient who had to have his own removed because of cancer.

The scaffold here instructed stem cells to create the windpipes lining, essentially using the body as an incubator to help direct their fate. This time, the UCL team utilised a cocktail of chemicals to help push the stem cells to make cartilage, so it remains to be seen if the scaffold will similarly drive ear cartilage formation once placed in the body.

What is clear, however, is that the field of tissue engineering is progressing at a remarkable pace and tailor-made tissues to treat a range of conditions are a real possibility in the near future."

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Commentary: field of tissue engineering is progressing at remarkable pace

PUBLIC RELEASE DATE:

19-Feb-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 19, 2014Joseph C. Glorioso, III, PhD (University of Pittsburgh School of Medicine, PA) devoted much of his research career to developing herpes viruses as efficient vectors for delivering therapeutic genes into cells. In recognition of his leadership and accomplishments, he has received a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Human Gene Therapy is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel* and publishing a Pioneer Perspective by each of the award recipients. The Perspective by Dr. Glorioso is available on the Human Gene Therapy website.

As he recounts in his essay "Herpes Simplex Viral Vectors: Late Bloomers with Big Potential," it took 30 years to create broadly applicable HSV vector designs and a useful gene delivery platform. Since herpes simplex virus has a natural affinity for the nervous system, Dr. Glorioso believes that "gene delivery to the brain represents the most important frontier for HSV-mediated gene therapy and provides a unique opportunity to study complex processes such as learning and memory and to treat complex genetic and acquired diseases, including brain degeneration, epilepsy, and cancer."

In addition, says Dr. Glorioso, some herpes viral delivery systems are proving useful for gene transfer in the emerging field of cellular reprogramming to produce stem cells for tissue regeneration.

"Joe began his work in gene therapy early in the development of the field focusing on the very challenging objective of targeting the central nervous system. His work with HSV vectors represents an incredibly elegant blending of basic virology and translational science," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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*The blue ribbon panel of leaders in cell and gene therapy, led by Chair Mary Collins, PhD, MRC Centre for Medical Molecular Virology, University College London selected the Pioneer Award recipients. The Award Selection Committee selected scientists that had devoted much of their careers to cell and gene therapy research and had made a seminal contribution to the field--defined as a basic science or clinical advance that greatly influenced progress in translational research.

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Joseph Glorioso, Ph.D., receives Pioneer Award

Precise and easy ways to rewrite human genes could finally provide the tools that researchers need to understand and cure some of our most deadly genetic diseases.

Over the last decade, as DNA-sequencing technology has grown ever faster and cheaper, our understanding of the human genome has increased accordingly. Yet scientists have until recently remained largely ham-fisted when theyve tried to directly modify genes in a living cell. Take sickle-cell anemia, for example. A debilitating and often deadly disease, it is caused by a mutation in just one of a patients three billion DNA base pairs. Even though this genetic error is simple and well studied, researchers are helpless to correct it and halt its devastating effects.

Now there is hope in the form of new genome-engineering tools, particularly one called CRISPR. This technology could allow researchers to perform microsurgery on genes, precisely and easily changing a DNA sequence at exact locations on a chromosome. Along with a technique called TALENs, invented several years ago, and a slightly older predecessor based on molecules called zinc finger nucleases, CRISPR could make gene therapies more broadly applicable, providing remedies for simple genetic disorders like sickle-cell anemia and eventually even leading to cures for more complex diseases involving multiple genes. Most conventional gene therapies crudely place new genetic material at a random location in the cell and can only add a gene. In contrast, CRISPR and the other new tools also give scientists a precise way to delete and edit specific bits of DNAeven by changing a single base pair. This means they can rewrite the human genome at will.

It is likely to be at least several years before such efforts can be developed into human therapeutics, but a growing number of academic researchers have seen some preliminary success with experiments involving sickle-cell anemia, HIV, and cystic fibrosis (see table below). One is Gang Bao, a bioengineering researcher at the Georgia Institute of Technology, who has already used CRISPR to correct the sickle-cell mutation in human cells grown in a dish. Bao and his team started the work in 2008 using zinc finger nucleases. When TALENs came out, his group switched quickly, says Bao, and then it began using CRISPR when that tool became available. While he has ambitions to eventually work on a variety of diseases, Bao says it makes sense to start with sickle-cell anemia. If we pick a disease to treat using genome editing, we should start with something relatively simple, he says. A disease caused by a single mutation, in a single gene, that involves only a single cell type.

In little more than a year, CRISPR has begun reinventing genetic research.

Bao has an idea of how such a treatment would work. Currently, physicians are able to cure a small percentage of sickle-cell patients by finding a human donor whose bone marrow is an immunological match; surgeons can then replace some of the patients bone marrow stem cells with donated ones. But such donors must be precisely matched with the patient, and even then, immune rejectiona potentially deadly problemis a serious risk. Baos cure would avoid all this. After harvesting blood cell precursors called hematopoietic stem cells from the bone marrow of a sickle-cell patient, scientists would use CRISPR to correct the defective gene. Then the gene-corrected stem cells would be returned to the patient, producing healthy red blood cells to replace the sickle cells. Even if we can replace 50 percent, a patient will feel much better, says Bao. If we replace 70 percent, the patient will be cured.

Though genome editing with CRISPR is just a little over a year old, it is already reinventing genetic research. In particular, it gives scientists the ability to quickly and simultaneously make multiple genetic changes to a cell. Many human illnesses, including heart disease, diabetes, and assorted neurological conditions, are affected by numerous variants in both disease genes and normal genes. Teasing out this complexity with animal models has been a slow and tedious process. For many questions in biology, we want to know how different genes interact, and for this we need to introduce mutations into multiple genes, says Rudolf Jaenisch, a biologist at the Whitehead Institute in Cambridge Massachusetts. But, says Jaenisch, using conventional tools to create a mouse with a single mutation can take up to a year. If a scientist wants an animal with multiple mutations, the genetic changes must be made sequentially, and the timeline for one experiment can extend into years. In contrast, Jaenisch and his colleagues, including MIT researcher Feng Zhang (a 2013 member of our list of 35 innovators under 35), reported last spring that CRISPR had allowed them to create a strain of mice with multiple mutations in three weeks.

Genome GPS

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CRISPR is the technology that could allow researchers to perform microsurgery on genes

Image Caption: Beating-heart cells derived from iPS cells are shown. A single DNA base-pair of the PRKAG2 gene was edited using the method developed by Drs. Miyaoka and Conklin. Credit: Luke Judge/Gladstone Institutes

Anne D. Holden, PhD Gladstone Institutes

Gladstones innovative technique in stem cells to boost scientists ability to study and potentially cure genetic disease

Sometimes biology is cruel. Sometimes simply a one-letter change in the human genetic code is the difference between health and a deadly disease. But even though doctors and scientists have long studied disorders caused by these tiny changes, replicating them to study in human stem cells has proven challenging. But now, scientists at the Gladstone Institutes have found a way to efficiently edit the human genome one letter at a time not only boosting researchers ability to model human disease, but also paving the way for therapies that cure disease by fixing these so-called bugs in a patients genetic code.

Led by Gladstone Investigator Bruce Conklin, MD, the research team describes in the latest issue of Nature Methods how they have solved one of science and medicines most pressing problems: how to efficiently and accurately capture rare genetic mutations that cause disease as well as how to fix them. This pioneering technique highlights the type of out-of-the-box thinking that is often critical for scientific success.

Advances in human genetics have led to the discovery of hundreds of genetic changes linked to disease, but until now weve lacked an efficient means of studying them, explained Dr. Conklin. To meet this challenge, we must have the capability to engineer the human genome, one letter at a time, with tools that are efficient, robust and accurate. And the method that we outline in our study does just that.

One of the major challenges preventing researchers from efficiently generating and studying these genetic diseases is that they can exist at frequencies as low as 1%, making the task of finding and studying them labor-intensive.

For our method to work, we needed to find a way to efficiently identify a single mutation among hundreds of normal, healthy cells, explained Gladstone Research Scientist Yuichiro Miyaoka, PhD, the papers lead author. So we designed a special fluorescent probe that would distinguish the mutated sequence from the original sequences. We were then able to sort through both sets of sequences and detect mutant cellseven when they made up as little one in every thousand cells. This is a level of sensitivity more than one hundred times greater than traditional methods.

The team then applied these new methods to induced pluripotent stem cells, or iPS cells. These cells, derived from the skin cells of human patients, have the same genetic makeup including any potential disease-causing mutations as the patient. In this case, the research team first used a highly advanced gene-editing technique called TALENs to introduce a specific mutation into the genome. Some gene-editing techniques, while effective at modifying the genetic code, involve the use of genetic markers that then leave a scar on the newly edited genome. These scars can then affect subsequent generations of cells, complicating future analysis. Although TALENs, and other similarly advanced tools, are able to make a clean, scarless single letter edits, these edits are very rare, so that new technique from the Conklin lab is needed.

Our method provides a novel way to capture and amplify specific mutations that are normally exceedingly rare, said Dr. Conklin. Our high-efficiency, high-fidelity method could very well be the basis for the next phase of human genetics research.

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Engineering The Human Genome One Letter At A Time