New Project Aims to Set Effectiveness Guidelines for Hemophilia Gene Therapy Trials – Hemophilia News Today
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New Project Aims to Set Effectiveness Guidelines for Hemophilia Gene Therapy Trials
Hemophilia News Today “With a growing pipeline of gene therapy products for hemophilia, it is an ideal time for this work,” Sean Tunis, president and CEO of CMTP, said in a news release. “This effort will potentially serve as a model for achieving consensus around outcomes … |
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New mechanism for genome regulation discovered
Science Daily “Gene therapy, or any treatment that relies on tight regulation of gene expression, could be improved by precisely targeting molecules to the right place in the nucleus,” says Karpen. “It is very difficult to target genes located in heterochromatin … |
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Stem Cells Try, But Fail, to Repair ACL Injuries
Newswise (press release) Newswise — The human body has a stockpile of stem cells, armed and ready to respond and repair damage to a variety of cells and tissues. Researchers have known heart attacks and strokes summon these cells, but, scientists recently discovered other … |
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Sarepta and Genethon Collaborate on Gene Therapy Research for Duchenne
Rare Disease Report Unlike Sarepta’s current pipeline that is focused on specific dystrophin gene mutations, Genethon’s micro-dystrophin gene therapy approach can target most patients with DMD, and the company has demonstrated proof-of-concept via robust gene expression … |
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AveXis’ Gene Therapy AVXS-101 Edges Closer to Pivotal Trial for SMA Type 1
SMA News Today AveXis is working with the U.S. Food and Drug Administration (FDA) to overcome final regulatory hurdles before starting a pivotal trial of AVXS-101 for the treatment of spinal muscular atrophy type 1 (SMA type 1) later in 2017. AVXS-101 is a gene … |