Category Archives: Gene Medicine

Penn Medicine researchers have discovered that hypermethylation -- the epigenetic ability to turn down or turn off a bad gene implicated in 10 to 30 percent of patients with Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Degeneration (FTD) -- serves as a protective barrier inhibiting the development of these diseases. Their work, published this month in Neurology, may suggest a neuroprotective target for drug discovery efforts.

"This is the first epigenetic modification of a gene that seems to be protective against neuronal disease," says lead author Corey McMillan, PhD, research assistant professor of Neurology in the Frontotemporal Degeneration Center in the Perelman School of Medicine at the University of Pennsylvania. Expansions in the offending gene, C9orf72, have been linked with TAR DNA binding protein (TDP-43) which is the pathological source that causes ALS and FTD.

"Understanding the role of C9orf72 has the possibility to be truly translational and improve the lives of patients suffering from these devastating diseases," says senior author, Edward Lee, MD, PhD, assistant professor of Neuropathology in Pathology and Laboratory Medicine at Penn.

McMillan and team evaluated 20 patients recruited from both the FTD Center and the ALS Center at the University of Pennsylvania who screened positive for a mutation in the C9orf72 gene and were clinically diagnosed with FTD or ALS. All patients completed a neuroimaging study, a blood test to evaluate C9orf72 methylation levels, and a brief neuropsychological screening assessment. The study also included 25 heathy controls with no history of neurological or psychiatric disease.

MRI revealed reduced grey matter in several regions that were affected in patients compared to controls. Grey matter is needed for the proper function of the brain in regions involved with muscle control, memory, emotions, speech and decision-making. Critically, patients with hypermethylation of C9orf72 showed more dense grey matter in the hippocampus, frontal cortex, and thalamus, regions of the brain important for the above described tasks and affected in ALS and FTD, suggesting that hypermethylation is neuroprotective in these regions.

To validate these findings, the Penn team also looked at autopsies of 35 patients with C9orf72 expansions and found that their pathology also suggested that increased methylation was associated with reduced neuronal loss in both the frontal cortex and hippocampus.

Longitudinal analysis was performed in 11 of the study patients to evaluate the neuroprotective effects of hypermethylation in individuals over their disease course. This showed reduced changes in grey matter of the hippocampus, thalamus, and frontal cortex, associated with hypermethlation suggesting that disease progresses more slowly over time in individuals with C9orf72 hypermethylation. Longitudinal neuropsychological assessments also showed a correlation between protected memory decline and hypermethylation.

These findings are consistent with a growing number of studies which have suggested the neuroprotective effects of the hypermethylation of C9orf72. "We believe that this work provides additional data supporting the notion that C9orf72 methylation is neuroprotective and therefore opens up the exciting possibility of a new avenue for precision medicine treatments and targets for drug development in neurodegenerative disease," says McMillan.

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The above story is based on materials provided by Perelman School of Medicine at the University of Pennsylvania. Note: Materials may be edited for content and length.

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Potential New Drug Target for Protection against Certain Neurodegenerative Diseases

Nobel Prize winners raise alarm over genetic engineering of humans.

A group of senior American scientists and ethics experts is calling for debate on the gene-engineering of humans, warning that technology able to change the DNA of future generations is now imminent.

In policy recommendations published today in the journal Science, eighteen researchers, including two Nobel Prize winners, say scientists should accept a self-imposed moratorium on any attempt to create genetically altered children until the safety and medical reasons for such a step can be better understood.

The concern is over a rapidly advancing gene-editing technology, called CRISPR-Cas9, which is giving scientists the ability to easily alter the genome of living cells and animals (see Genome Surgery). The same technology could let scientists correct DNA letters in a human embryo or egg cell, for instance to create children free of certain disease-causing genes, or perhaps with improved genetics.

What we are trying to do is to alert people to the fact that this is now easy, says David Baltimore, a Nobel Prize winner and former president of Caltech, and an author of the letter. We cant use the cover we did previously, which is that it was so difficult that no one was going to do it.

Many countries already ban germ line engineeringor changing genes in a way that would be heritable from one generation to the nextonethical or safety grounds. Others, like the U.S., have strict regulations that would delay the creation of gene-edited children for years, if not decades. But some countries have weak rules, or none at all, and Baltimore said a reason scientists were speaking publicly now was to keep people from doing anything crazy.

The advent of CRISPR is raising social questions of a kind not confronted since the 1970s, when the ability to change DNA in microrganisms was first developed. In a now famous meeting in 1975, in Asilomar, California, researchers agreed to avoid certain kinds of experiments that were then deemed dangerous. Baltimore, who was one of the organizers of the Asilomar meeting, says the scientists behind the letter want to offer similar guidance for gene-engineered babies.

The prospect of genetically modified humans is surprisingly close at hand. A year ago, Chinese researchers created monkeys whose DNA was edited using CRISPR (see 10 Breakthrough Technologies 2014: Genome Editing).

Since then,several teams of researchers in China, the U.S., and the U.K. have begun using CRISPR to change the DNA of human embryos, eggs, and sperm cells, with an eye toward applying the technology at in vitro fertility (IVF) clinics. That laboratory research was described by MIT Technology Review earlier this month (see Engineering the Perfect Baby).

Last week, in Nature, representatives of an industry group, the Alliance for Regenerative Medicine, recommended a wider moratorium that would also include a cessation of such laboratory studies, which it termed dangerous and ethically unacceptable (see Industry Body Calls for Gene-Editing Moratorium).

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Scientists Call for a Summit on Gene-Edited Babies

IMAGE:Kazuhiro Nitta, Ph.D., and his colleagues at Karolinska Institutet have shown that the gene regulatory code has remained unchanged across millions of years of evolution. view more

Credit: Karolinska Institutet

The language used in the switches that turn genes on and off has remained the same across millions of years of evolution, according to a new study led by researchers at Karolinska Institutet in Sweden. The findings, which are published in the scientific journal "eLife", indicate that the differences between animals reside in the content and length of the instructions that are written using this conserved language.

Tiny fruit flies look very different from humans, but both are descended from a common ancestor that existed over 600 million years ago. Differences between animal species are often caused by the same or similar genes being switched on and off at various times and in different tissues in each species.

Each gene has a regulatory region that contains the instructions controlling when and where the gene is expressed. These instructions are written in a language often referred to as the 'gene regulatory code'. This code is read by proteins called transcription factors that bind to specific 'DNA words' and either increase or decrease the expression of the associated gene.

The gene regulatory regions differ between species. However, until now, it has been unclear if the instructions in these regions are written using the same gene regulatory code, or whether transcription factors found in different animals recognise different DNA words.

In the current study, the researchers used high throughput methods to identify the DNA words recognised by more than 240 transcription factors of the fruit fly, and then developed computational tools to compare them with the DNA words of humans.

"We observed that, in spite of more than 600 million years of evolution, almost all known DNA words found in humans and mice were recognised by fruit fly transcription factors", says Kazuhiro Nitta at the Department of Biosciences and Nutrition at Karolinska Institutet, first author of the study.

The researchers also noted that both fruit flies and humans have a few transcription factors that recognise unique DNA words and confer properties that are specific to each species, such as the fruit fly wing. Likewise, transcription factors that exist only in humans operate in cell types that do not exist in fruit flies. The findings suggest that changes in transcription factor specificities contribute to the formation of new types of cells.

The study of fundamental properties of gene switches is important in medicine, as faulty gene switches have been linked to many common diseases, including cancer, diabetes and heart disease. The research was funded by, among others, Center for Innovative Medicine at Karolinska Institutet and Gran Gustafsson Foundation.

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Language of gene switches unchanged across the evolution

SAN DIEGO, CA--(Marketwired - March 17, 2015) - Targazyme, Inc., a clinical stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for a variety of cell therapies including stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted IND Clearance to M.D. Anderson Cancer Center to start enrolling patients in a Phase I/II clinical study to evaluate the safety and efficacy of TZ101-fucosylated regulatory T cells (Tregs) in preventing and reducing the severity and incidence of graft vs. host disease (GVHD) in patients eligible for hematologic stem cell transplant.

GVHD is a serious, life-threating complication of stem cell transplantation, affecting over 60% of all patients undergoing allogeneic stem cell transplantation.This clinical study hopefully will affirm Targazyme's novel treatment approach.TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a high percentage of these patients and is very difficult to manage clinically.If the efficacy of TZ101-treated Tregs is demonstrated in GVHD, then the company expects the treatment to be useful in a number of autoimmune diseases such as rheumatoid arthritis, multiple sclerosis and others.

"This Phase I/II study will hopefully translate into the clinic and give our patients the impressive increases in survival seen in animal models and also the persistence/activity of TZ101-treated Tregs in the preclinical models of GVHD," said Dr. Lynne A. Bui, Senior Vice President of Development at Targazyme.

"This FDA IND clearance for TZ101-treated regulatory T cells for the prevention and treatment of GVHD provides additional validation of our innovative products to potentially improve efficacy outcomes for patients undergoing stem cell transplantation and immunotherapy," said Lynnet Koh, CEO and Chairman of the Board of Targazyme."We are excited that the study is open for enrollment at M.D. Anderson Cancer Center to start treating patients with this promising modality for preventing GVHD in patients undergoing stem cell transplantation."

About Targazyme, Inc.

Targazyme, Inc. is a San Diego based, clinical stage biopharmaceutical company developing novel enzyme-based platform technologies and products to improve clinical efficacy outcomes for stem cell medicine, immunotherapy for cancer and autoimmune diseases, gene therapy and regenerative medicine.

In addition to conducting immunotherapy proof of concept trials, Targazyme is also gearing up for its global product registration trial in hematopoietic stem cell transplantation.The company's clinical-grade fucosyltransferase enzymes and small molecule products (TZ101 and TZ102) are off-the-shelf kits used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice.The company has received a number of world-wide patents, multiple FDA orphan drug designations and major medical/scientific awards and grants.

Targazyme has partnerships and collaborations with Kyowa Hakko Kirin and Florida Biologix, as well as various medical research institutions including The University of Texas M.D. Anderson Cancer Center, Oklahoma Medical Research Foundation, Texas Transplant Institute, Case Western/University Hospitals, Scripps Hospitals, Fred Hutchinson Cancer Research Center, University of California Los Angeles Medical Center, Stanford University Medical Center, University of Minnesota Medical Center, University of California San Diego Medical Center, Sanford-Burnham Medical Research Institute, Indiana University, Memorial Sloan Kettering Cancer Center, and New York Blood Center.Learn more at http://www.targazyme.com.

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Targazyme, Inc. and M.D. Anderson Cancer Center Receive FDA IND Clearance for Phase I/II Study of TZ101-Treated ...

Gene-editing companies say research on altering the DNA of human reproductive cells is dangerous and unethical.

Officials of a biotechnology industry group have called for a voluntary moratorium on using new DNA-editing techniques to change the genetic characteristics of human embryos in laboratory research.

In an editorial published today by the journal Nature, Edward Lanphier, CEO of the biotechnology company Sangamo Biosciences, and four colleagues write that scientists should agree not to modify the DNA of human reproductive cells because it raises safety and ethical risks including the danger of unpredictable effects on future generations.

New gene-editing techniques, in particular one called CRISPR, have given scientists powerful and useful new ways to swap and change DNA letters inside of living cells for the first time (see Genome Surgery).

Recently, some scientific teams have started to study whether CRISPR would be able to correct disease genes in future generations of peoplefor instance, by repairing genes during in vitro fertilization, or in eggs or sperm. The idea of such germ line modification would be to install healthy versions of genes, which children would be born with.

The emergence of active research around germ-line editing, which is taking place in China, at Harvard University, and at a publicly traded biotechnology company called OvaScience, were described last week by MIT Technology Review (see Engineering the Perfect Baby).

But the idea of using editing technology to improve children is as controversial as it is medically powerful. In their editorial, Lanphier, whose coauthors include Fyodor Urnov, co-developer of a different gene-editing system, raise the concern that such techniques might be exploited for non-therapeutic modifications. That could mean, for instance, changing the physical traits of children.

The availability of technology to carry out genetic engineering in human germ-line cells is driving intense debate in scientific circles and may eventually become a legal issue in the United States and other countries.

The authors call for a cessation of basic research is unusual and likely to be opposed by scientists as an intrusion on the quest for scientific knowledge.

George Church, a professor at Harvard Medical School whose laboratory studies CRISPR and germ-line editing, says a voluntary moratorium would be weak compared with existing regulations that nearly all countries impose on the use of new medical technologies until they are proven safe and effective in animals or human [tests]. Church was referring to rules governing the birth of actual gene-edited children, not basic research.

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Industry Body Calls for Gene-Editing Moratorium

A call by scientists to halt to precision gene-editing of DNA in human embryos would allow time to work out safety and ethical issues

Sperm cell fertilizing an egg. Credit: Wikimedia Commons

Amid rumors that precision gene-editing techniques have been used to modify the DNA of human embryos, researchers have called for a moratorium on the use of the technology in reproductive cells.

In a Comment published on March 12 inNature, Edward Lanphier, chairman of the Alliance for Regenerative Medicine in Washington DC, and four co-authors call on scientists to agree not to modify human embryos even for research.

Such research could be exploited for non-therapeutic modifications. We are concerned that a public outcry about such an ethical breach could hinder a promising area of therapeutic development, write Lanphier and his colleagues, who include Fyodor Urnov, a pioneer in gene-editing techniques and scientist at Sangamo BioSciences in Richmond, California. Many groups, including Urnov's company, are already using gene-editing tools to develop therapies that correct genetic defects in people (such as by editing white blood cells). They fear that attempts to produce designer babies by applying the methods to embryos will create a backlash against all use of the technology.

Known as germline modification, edits to embryos, eggs or sperm are of particular concern because a person created using such cells would have had their genetic make-up changed without consent, and would permanently pass down that change to future generations.

We need a halt on anything that approaches germline editing in human embryos, Lanphier, who is also chief executive of Sangamo, toldNatures news team.

But other scientists disagree with that stance. Although there needs to be a wide discussion of the safety and ethics of editing embryos and reproductive cells, they say, the potential to eliminate inherited diseases means that scientists should pursue research.

Related trials Geneticist Xingxu Huang of ShanghaiTech University in China, for example, is currently seeking permission from his institutions ethics committee to try genetically modifying discarded human embryos. In February 2014, he reportedusing a gene-editing technique to modify embryos that developed into live monkeys. Human embryos would not be allowed to develop to full term in his experiments, but the technique gives lots of potential for its application in humans, he says.

Besides Huangs work, gene-editing techniques are also being used by Juan Carlos Izpisua Belmonte, a developmental biologist at the Salk Institute for Biological Studies in La Jolla, California, to eliminate disease-causing mutations from mitochondria, the cell's energy-processing structures. Belmonte's work is on unfertilized eggs; human eggs with such modified mitochondria could one day be used inin vitrofertilization (IVF) procedures to prevent a woman's offspring from inheriting mitochondrial disease.

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DNA Editing of Human Embryos Alarms Scientists

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Newswise Researchers at the University of California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimers disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from adult stem cells.

Our results suggest that certain gene variants allow us to reduce the amount of beta amyloid produced by neurons, said senior author Lawrence Goldstein, PhD, director of UC San Diego Sanford Stem Cell Clinical Center and UC San Diego Stem Cell Program. This is potentially significant for slowing the progression of Alzheimers disease. AD is the most common cause of dementia in the United States, afflicting one in nine people age 65 and older.

The genetic risk factor investigated are variants of the SORL1 gene. The gene codes for a protein that affects the processing and subsequent accumulation of beta amyloid peptides, small bits of sticky protein that build up in the spaces between neurons. These plaques are linked to neuronal death and related dementia.

Previous studies have shown that certain variants of the SORL1 gene confer some protection from AD, while other variants are associated with about a 30 percent higher likelihood of developing the disease. Approximately one-third of the U.S. adult population is believed to carry the non-protective gene variants.

The studys primary finding is that variants in the SORL1 gene may also be associated with how neurons respond to a natural compound in the brain that normally acts to protect nerve cell health. The protective compound, called BDNF, short for brain-derived neurotrophic factor, is currently being investigated as a potential therapy for a number of neurological diseases, including AD, because of its role in promoting neuronal survival.

For the study, UC San Diego researchers took skin cells from 13 people, seven of whom had AD and six of whom were healthy control subjects, and reprogrammed the skin cells into stem cells. These stem cells were coaxed to differentiate into neurons, and the neurons were cultured and then treated with BDNF.

The experiments revealed that neurons that carried disease-protective SORL1 variants responded to the therapy by reducing their baseline rate of beta amyloid peptide production by, on average, 20 percent. In contrast, the neurons carrying the risk variants of the gene, showed no change in baseline beta amyloid production.

BDNF is found in everyones brain, said first author Jessica Young, PhD, a postdoctoral fellow in the Goldstein laboratory. What we found is that if you add more BDNF to neurons that carry a genetic risk factor for the disease, the neurons dont respond. Those with the protective genetic profile do.

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Boosting A Natural Protection Against Alzheimer's Disease

Researchers at the University of Bonn discover a new hereditary factor associated with a rare disease

IMAGE:This image shows Prof. Dr. Michael Ludwig, Dr. Heiko Reutter and Prof. Dr. Markus Nthen of the University of Bonn Hospital (from left). view more

Credit: (c) Photo: Katharina Wislsperger/UKB

An interdisciplinary team of researchers under the direction of the University of Bonn Hospital have discovered a gene which is associated with a rare congenital anomaly of the urinary tract called classic bladder exstrophy. It increases the likelihood that the urinary tract will not form properly during embryonic development. The finding is an important step for understanding the development of urinary tract malformations in general and for developing prophylactic measures. The results are published in the current online edition of the journal "PloS Genetics".

The kidneys and urinary tract are the sites affected most frequently by congenital malformations. Approximately 1 out of every 200 children suffers from such a malformation. "These diseases make up about 20 to 30 percent of all congenital malformations," says Associate Professor Dr. Heiko Reutter from the Institute of Human Genetics and the Department of Neonatology and Pediatric Intensive Care Medicine of the University of Bonn.

For many years, the pediatrician has investigated the genetic causes of classic bladder exstrophy comprising malformations ranging from the bladder to the entire urinary tract. These malformations frequently result in urinary tract infections, incontinence, renal damage and sexual dysfunction. Approximately one out of 20,000 newborns is affected by this rare disease which is considered to be one of the most severe forms of malformations on this spectrum. "Congenital classic bladder exstrophy thus represents an enormous challenge in the medical care of patients affected and their families," says Dr. Reutter.

Focus at the Center for Rare Diseases

To date, the genetic causes of this rare disease have been basically unknown. In the past ten years, with the bladder extrophy/epispadias self-help group and leading pediatric urologists and pediatric surgeons in Germany - including from the Barmherzigen Brder Pediatric Hospital in Regensburg as well as the universities of Mainz and Ulm - researchers at the University of Bonn hospital have been able to gather the largest group of patients in the world. The researchers in Bonn received additional support for the current study from researchers at the Max Planck Institute for Molecular Genetics in Berlin. Assistance was also provided by the Center for Rare Diseases at the University of Bonn Hospital (ZSEB). The researchers focus on rare uro-rectal malformations there.

Using blood samples from a total of 210 patients, the scientists isolated the genetic information and compared it with a control group of healthy persons. The researchers used automated analysis methods to record more than 700,000 genetic markers in each case which are evenly distributed throughout the DNA. The evaluation using biostatistical methods revealed a clear connection with an altered gene: ISL1, which is located on chromosome five. "In this way, a gene in connection with this disease was identified for the very first time," says Prof. Dr. Michael Ludwig from the Institute of Clinical Chemistry and Clinical Pharmacology of the University of Bonn Hospital.

The search for other genes

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Gene leads to malformation of the urinary tract

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. Thus far, the new therapy has proven safe.

"The result was stunning," said Timothy Nichols, MD, director of the Francis Owen Blood Research Laboratory at the University of North Carolina School of Medicine and co-senior author of the paper. "Just a small amount of new factor IX necessary for proper clotting produced a major reduction in bleeding events. It was extraordinarily powerful."

The idea behind gene therapy is that doctors could give hemophilia patients a one-time dose of new clotting genes instead of a lifetime of multiple injections of recombinant factor IX that until very recently had to be given several times a week. A new FDA-approved hemophilia treatment lasts longer than a few days but patients still require injections at least once or twice a month indefinitely.

This new gene therapy approach, like other gene therapy approaches, would involve a single injection and could potentially save money while providing a long-term solution to a life-long condition. A major potential advantage of this new gene therapy approach is that it uses lentiviral vectors, to which most people do not have antibodies that would reject the vectors and make the therapy less effective.

In human clinical studies, approximately 40 percent of the potential participants screened for a different kind of viral vector -- called adeno-associated viral vectors -- have antibodies that preclude them from entering AAV trials for hemophilia gene therapy treatment. This means that more people could potentially benefit from the lentivirus gene therapy approach.

Hemophilia is a bleeding disorder in which people lack a clotting factor, which means they bleed much more easily than people without the disease. Often, people with hemophilia bleed spontaneously into joints, which can be extremely painful and crippling. Spontaneous bleeds into soft tissues are also common and can be fatal if not treated promptly. Hemophilia A affects about one in 5,000 male births. These patients do not produce enough factor VIII in the liver. This leads to an inability to clot. Hemophilia B affects about one in 35,000 births; these patients lack factor IX.

This new method was spearheaded by Luigi Naldini, PhD, director of the San Raffaele Telethon Institute for Gene Therapy and co-senior author on the Science Translational Medicine paper.

For this study, Naldini and Nichols developed a way to use a lentivirus, which is a large retrovirus, to deliver factor IX genes to the livers of three dogs that have naturally occurring hemophilia. The researchers removed the genes involved in viral replication. "Essentially, this molecular engineering rendered the virus inert," Nichols said. "It had the ability to get into the body but not cause disease." This process turned the virus into a vector -- simply a vehicle to carry genetic cargo.

Unlike some other viral vectors that have been used for gene therapy experiments, the lentiviral vector is so large that it can carry a lot of payload -- namely, the factor IX genes that people with hemophilia B lack. (This approach could also be used for hemophilia A where the FVIII gene is considerably larger.)

These viral vectors were then injected directly into the liver or intravenously. After more than three years, the three dogs in the study experienced zero or one serious bleeding event each year. Before the therapy, the dogs experienced an average of five spontaneous bleeding events that required clinical treatment. Importantly, the researchers detected no harmful effects.

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New gene therapy for hemophilia shows potential as safe treatment

Amid rumours that precision gene-editing techniques have been used to modify the DNA of human embryos, researchers have called for a moratorium on the use of the technology in reproductive cells.

MOLEKUUL/SCIENCE PHOTO LIBRARY

The gene-editing technique CRISPR uses an enzyme (white) and RNA guides (blue) to cut DNA at a point specified by a DNA fragment (red).

In a Comment published on 12 March in Nature1, Edward Lanphier, chairman of the Alliance for Regenerative Medicine in Washington DC, and four co-authors call on scientists to agree not to modify human embryos even for research.

Such research could be exploited for non-therapeutic modifications. We are concerned that a public outcry about such an ethical breach could hinder a promising area of therapeutic development, write Lanphier and his colleagues, who include Fyodor Urnov, a pioneer in gene-editing techniques and scientist at Sangamo BioSciences in Richmond, California. Many groups, including Urnov's company, are already using gene-editing tools to develop therapies that correct genetic defects in people (such as by editing white blood cells). They fear that attempts to produce designer babies by applying the methods to embryos will create a backlash against all use of the technology.

Known as germline modification, edits to embryos, eggs or sperm are of particular concern because a person created using such cells would have had their genetic make-up changed without consent, and would permanently pass down that change to future generations.

We need a halt on anything that approaches germline editing in human embryos, Lanphier, who is also chief executive of Sangamo, told Natures news team.

But other scientists disagree with that stance. Although there needs to be a wide discussion of the safety and ethics of editing embryos and reproductive cells, they say, the potential to eliminate inherited diseases means that scientists should pursue research.

Geneticist Xingxu Huang of ShanghaiTech University in China, for example, is currently seeking permission from his institutions ethics committee to try genetically modifying discarded human embryos. In February 2014, he reported2 using a gene-editing technique to modify embryos that developed into live monkeys. Human embryos would not be allowed to develop to full term in his experiments, but the technique gives lots of potential for its application in humans, he says.

Besides Huangs work, gene-editing techniques are also being used by Juan Carlos Izpisua Belmonte, a developmental biologist at the Salk Institute for Biological Studies in La Jolla, California, to eliminate disease-causing mutations from mitochondria, the cell's energy-processing structures. Belmonte's work is on unfertilized eggs; human eggs with such modified mitochondria could one day be used in in vitro fertilization (IVF) procedures to prevent a woman's offspring from inheriting mitochondrial disease.

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Scientists sound alarm over DNA editing of human embryos