Monthly Archives: September 2014

One of the inventors of gene editing says scientists should proceed cautiously before testing it in people.

Feng Zhang

Citing the risk of deadly mistakes, a leading researcher speaking at MIT Technology ReviewsEmTech conferenceon Tuesday said the risks of gene editing need to be better understood before the technology can be used in medical studies.

Feng Zhang, a researcher at MIT, helped invent a powerful new way to alter DNA that he compared in his talk to a search-and-replace function for the genome.

Several startups have already sprung up to turn the technology into new kinds of gene-therapy drugs, including CRISPR Therapeutics and Editas Medicine, a biotechnology company that Zhang cofounded last year with venture capitalists who invested $43 million.

These companies hope to correct diseases, like cystic fibrosis, caused by faulty DNA. In other cases, Zhang said, changing a persons DNA could provide a protective effectfor instance, conferring immunity to HIV.

The concept is very powerful, but to make any correction in the body is very challenging, he said.

Looming over researchers is the 1999 death of Jesse Gelsinger, a volunteer in an early gene therapy study in Pennsylvania. That failure dealt a huge setback to genetic drugs. Later it was shown that such treatments, even when they work, could sometimes cause cancer by making unwanted changes to a persons genome.

One of the early lessons from gene therapy is to go slowly, said Zhang. The lesson is that we need to understand a system carefully before putting it into a person.

Gradually, however, gene therapy has staged a comeback. In 2012, a treatment called Glybera was the first to be approved in Europe. Its not yet for sale in the U.S., but numerous gene treatments are being tested in patients.

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EmTech: Risks of Gene-Editing Drugs Need Study, Pioneer Says

Posted by Jennifer Schutz (@jschutz) 6 day(s) ago

Mayo Clinic Center for Regenerative Medicine Forms Collaboration with National University Ireland Galway

ROCHESTER, Minn. The Mayo Clinic Center for Regenerative Medicine and colleagues at the National University Ireland Galway have signed a formal memorandum of understanding (MOU) to pave the way for joint clinical trials using regenerative therapies.

The MOU follows years of close collaboration with NUI Galways Regenerative Medicine Institute (REMEDI) and the Network of Excellence for Functional Biomaterials (NFB) and will focus on adult stem cell therapy, gene therapy, biomaterials and biomedical engineering. Furthermore, the agreement facilitates ongoing student and staff exchange between Galway and the United States.

MEDIA CONTACT:Jennifer Schutz, Mayo Clinic Public Affairs, 507-284-5005, newsbureau@mayo.edu

Journalists: Sound bites withDr. Windebankare in the downloads.

Anthony Windebank, M.D., deputy director for Discovery, Mayo Clinic Center for Regenerative Medicine, and Professor Timothy OBrien, director of the REMEDI, were among those present at the signing in Galway.

Both the National University Ireland Galway and the Mayo Clinic Center for Regenerative Medicine have laboratories which are compliant with current good manufacturing practice (GMP) regulations as it applies to cell manufacturing, says Professor OBrien. This allows us to initiate joint trials of regenerative therapies that will produce identical cell products.

The U.S. Food and Drug Administration and the European Medicines Agency are making efforts to streamline and facilitate introduction of new therapies on both sides of the Atlantic Ocean. Carrying out these approval processes and completing joint studies will facilitate more rapid introduction of new therapies for patients.

Pictured at the signing: Prof. Lokesh Joshi, Vice President for Research, NUI Galway; Dr. Jim Browne, President, NUI Galway; Dr. Anthony Windebank, Deputy Director for Discovery, Mayo Clinic Center for Regenerative Medicine; and Prof. Tim OBrien, Director of REMEDI NUI Galway.

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Mayo Clinic Center for Regenerative Medicine Forms Collaboration with National University Ireland Galway

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Newswise Philadelphia, Sept. 23, 2014 An international team of scientists has identified a gene mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells. Studying a family in which three generations had blood disorders, the researchers discovered a defect in a gene that regulates telomeres, chromosomal structures with crucial roles in normal cell function.

Identifying this causal defect may help suggest future molecular-based treatments that bypass the gene defect and restore blood cell production, said study co-leader Hakon Hakonarson, M.D., Ph.D., director of the Center for Applied Genomics at The Childrens Hospital of Philadelphia (CHOP).

Hakonarson and CHOP colleagues collaborated with Australian scientists on the study, published online Sept. 9 in the journal Blood.

Were thrilled by this discovery which has advanced our understanding of certain gene mutations and the causal relationship to specific diseases, said study co-leader Tracy Bryan, Ph.D., Unit Head of the Cell Biology Unit at the Childrens Medical Research Institute in Westmead, New South Wales, Australia.

The research team studied an Australian family with aplastic anemia and other blood disorders, including leukemia. Hakonarson and lead analyst Yiran Guo, Ph.D., along with genomics experts from BGI-Shenzhen, performed whole-exome sequencing on DNA from the families and identified an inherited mutation on the ACD gene, which codes for the telomere-binding protein TPP1.

Telomeres, complex structures made of DNA and protein, are located on the end of chromosomes, where they protect the chromosomes stability. They are sometimes compared to plastic tips at the end of shoelaces that prevent the laces from fraying.

Telomeres shorten after each cell division, and gradually lose their protective function. Aging cells, with their shortened telomeres, become progressively more vulnerable to DNA damage and cell death. Separately from the aging process, certain inherited and acquired disorders may shorten telomeres and injure rapidly dividing blood-forming cells produced in bone marrow. This leads to bone marrow failure, one example of which is aplastic anemia.

Bryans team investigated the function of the ACD gene. They determined that the mutation shortened telomeres and interrupted the ability of telomeres to attract the enzyme telomerase, which counteracts telomere shortening and thus protects cells.

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Gene Mutation Discovered in Blood Disorder