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A Simplified Immune Suppression Scheme Leads to Persistent Micro-dystrophin Expression in Duchenne Muscular Dystrophy Dogs

Authors
Journal
Human Gene Therapy
1043-0342
Publisher
Mary Ann Liebert
Publication Date
Volume
23
Issue
2
Identifiers
DOI: 10.1089/hum.2011.147
Keywords
  • Research Articles

Abstract

Shin and colleagues develop and test a novel immune suppression scheme to allow for successful recombinant adeno-associated virus (AAV)-mediated microdystrophin gene transfer to dystrophic dogs. This shorter, simplified scheme uses readily available drugs such as cyclosporine and mycophenolate, administered over the course of 5 weeks. Adult dystrophic dog limb muscles injected with AAV encoding a canine dystrophin microgene under the new immune suppression protocol show sustained transduction for nearly half a year.

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