making muscle wasting history

Progress in Gene Therapy for DMD: Slipping Full-Length Dystrophin Past the Immune System

• Last Edited: 15 Sep 2009
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The immune system is one of the biggest obstacles to gene therapy for Duchenne muscular dystrophy (DMD), but researchers have devised a new gene therapy method that can largely evade the body̵...

Tags: adenovirus, gene therapy.

Viral based Gene Therapy - A review by Dr Karl Bettelheim

• Last Edited: 30 Jul 2010
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Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009 IntroductionIn my first review, I listed possible treatments under three headings and to remind readers...

Tags: AAV serotypes, Gene Therapy.

Crossing the Valley of Despair- A review by Dr Karl Bettelheim

• Last Edited: 30 Jul 2010
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Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009Although there have been some very good studies published recently especially on the use of viruses to transfer human genes...

Tags: gene therapy, exonskipping, utrophin upregulation, immunodeficiency XSCID, EGFR gene, gefitinib.

Gene therapy in Muscular Dystrophy. - A review by Dr Karl Bettelheim

• Last Edited: 30 Jul 2010
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Article submitted by Dr Karl Bettelheim published on the 18th of July 2009There has recently appeared an excellent review (1) of current studies on gene therapy for muscular dystrophies. While poi...

Tags: Exonskipping, phosphorodiamidate morpholino oligomers, Gene therapy.