Current Neurology and Neuroscience Reports

, Volume 1, Issue 1, pp 89–96 | Cite as

Progress in gene therapy for Duchenne muscular dystrophy

  • Paula R. Clemens
  • F Jason Duncan
Article

Abstract

Gene transfer research for Duchenne muscular dystrophy (DMD) has brought the goal of successful treatment of this devastating, inherited disease closer to being a reality. Although gene therapeutic approaches for DMD patients are not yet in clinical use, recent advances using DMD animal models are encouraging. Progress in vector design, such as high-capacity adenoviral vectors, targeted adenoviral vectors, and heterodimerization of DNA delivered by adeno-associated virus (AAV) vectors have advanced the field considerably. The recent studies into the pharmacologic-induced read-through of stop codons, the increased study of utrophin and its upregulation, and the introduction of point mutation correction using chimeric oligonucleotides have expanded the field, providing new avenues of inquiry.

Keywords

Duchenne Muscular Dystrophy Adenoviral Vector Duchenne Muscular Dystrophy Dystrophin Gene Helper Virus 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.
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Copyright information

© Current Science Inc 2001

Authors and Affiliations

  • Paula R. Clemens
    • 1
  • F Jason Duncan
    • 1
  1. 1.Department of NeurologyUniversity of PittsburghPittsburghUSA

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