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Last updated on April 19, 2014 at 1:20 EDT

Latest Human Gene Therapy Stories

2012-05-08 15:27:53

Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in several articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website. After 20 years of promising research, testing in animal models, and initial clinical trial...

2011-12-13 14:58:05

A recent article in the New England Journal of Medicine showed that Factor IX gene therapy in patients with hemophilia B was able to convert severe hemophilia to moderate or even mild disease. In a timely commentary, Thierry VandenDriessche, PhD, Methods Editor for Human Gene Therapy and Human Gene Therapy Methods says that this clinical trial represents an important milestone and "demonstrates unequivocally that gene therapy can result in a sustained therapeutic effect in hemophilia B...

2010-10-07 13:43:07

The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com). The article, which is published online ahead of print, is available free online at www.liebertpub.com/hum The growing popularity in the gene therapy community of using recombinant...

2010-08-16 13:19:06

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com), describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free...