Sentences with phrase «most gene therapy studies»

In most gene therapy studies, a «normal» gene is inserted into the genome to replace an «abnormal,» disease - causing gene.

Not exact matches

«I've been doing this for more than 40 years and this is one of the most exciting developments we've seen,» says Jerry Mendell, MD, lead author of the study and director of the Center for Gene Therapy at Nationwide Children's.

UPenn gene therapy researcher Jean Bennett, who heads that study (which is separate from Jacobson's) and reported the most dramatic improvements in patients» vision, says those gains seem stable for as long as 7.5 years.

Recently published in the journal Oncotarget, this new study is the most recent in a long line of studies led by Paul B. Fisher, M.Ph., Ph.D., investigating the use of viral gene therapy to treat a variety of cancers.

Most recently, she has led the planning and execution of a program for a new gene therapy product candidate, from ideation through proof - of - concept study, and mapping out the development plan.

Led by Florian Eichler, MD, from Harvard Medical School and Massachusetts General Hospital, the study tested the first successful gene therapy treatment for cerebral adrenoleukodystrophy (ALD), a fatal degenerative brain disease that most severely affects boys.

«The ability to direct a gene to a specific cell type and prevent expression in other cell types is a powerful new tool that allows us to bypass one of the most troubling safety concerns facing gene therapy,» said Michael Parmacek, MD, assistant professor of medicine at the University of Chicago and director of the study.

«Our goal has been to expand the study of gene therapy beyond Type 1 infants to address the urgent medical needs of children with SMA Type 2, and we look forward to understanding the potential clinical impact of AVXS - 101 in these patients who, left untreated, will never walk on their own and most will never stand without assistance.»