Gene Therapy

Nature publishes results of gene therapy treatment in phase 1/2 beta-thalassemia study

Published in Gene Therapy Weekly, September 30th, 2010

Genetix Pharmaceuticals Inc.) an emerging leader in the development of innovative gene therapies for severe genetic disorders, announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin™ gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

The patient, who had been transfusion dependent since early childhood, has become transfusion independent for the past 21 months - more than two years after treatment with the LentiGlobin vector. The study also identified a subset of cells with the corrected beta-globin gene that...

Want to see the full article?

Purchase this article for only $3.00

We're a pay-per-view site for premium content. If you'd like to purchase this article, it's only $3.00.

Welcome to NewsRx!

Learn more about a six-week, no-risk free trial of Gene Therapy Weekly

Source: Gene Therapy Weekly (2010-09-30)

NewsRx is Social

Top Medical Newsletters

Awards

Best Health/Healthcare Content, 2012
Best Health/Healthcare Content, 2011
Best e-Business Site, 2010
Best e-Business Site, 2009
Best e-Business Site, 2008
Best e-Business Site, 2007
Best e-Business Site, 2006
Best Healthcare Content, 2005
Best Overall Internet Site, 2005
Best Interactive Site, 2005

Facts & Stats

NewsRx also is available at LexisNexis, Gale, ProQuest, Factiva, Dialog, Thomson Reuters, NewsEdge, and Dow Jones.

Google 2010 PageRank: #2 Among Top Health News and Media Publications
Google 2010 PageRank: #2 Among Top Science Publications in Biology/Physiology
Google 2010 PageRank: #2 Among Top News and Media for the Business of Pharmaceuticals
Amazon's Alexa 2010 PageRank: #2 News and Media Site for the Pharmaceutical Industry