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Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy.

Publication ,  Journal Article
Hauser, MA; Amalfitano, A; Kumar-Singh, R; Hauschka, SD; Chamberlain, JS
Published in: Neuromuscul Disord
July 1997

We have been exploring the feasibility of gene therapy for Duchenne muscular dystrophy by characterizing parameters important for the design of therapeutic protocols. These studies have used transgenic mice to analyze expression patterns of multiple dystrophin vectors, and have been accompanied by the development of viral vectors for gene transfer to dystrophic mdx mouse muscle. Analysis of transgenic mdx mice indicates that greater than 50% of the fibers in a muscle group must express dystrophin to prevent development of a significant dystrophy, and that low-level expression of truncated dystrophins can function very well. These results suggest that gene therapy of DMD will require methods to transduce the majority of fibers in critical muscle groups with vectors that express moderate levels of dystrophin proteins. Strategies for the development of viral vectors able to deliver dystrophin genes to muscle include the use of muscle specific regulatory sequences coupled with deletion of viral gene sequences to limit virus-induced immune rejection of transduced tissues. These strategies should enable production of adenoviral vectors expressing full-length dystrophin proteins in muscle.

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Published In

Neuromuscul Disord

DOI

ISSN

0960-8966

Publication Date

July 1997

Volume

7

Issue

5

Start / End Page

277 / 283

Location

England

Related Subject Headings

  • Virus Replication
  • Neurology & Neurosurgery
  • Mutagenesis, Insertional
  • Muscular Dystrophy, Animal
  • Muscles
  • Mice, Transgenic
  • Mice, Inbred mdx
  • Mice
  • Genetic Vectors
  • Genetic Therapy
 

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Hauser, M. A., Amalfitano, A., Kumar-Singh, R., Hauschka, S. D., & Chamberlain, J. S. (1997). Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy. Neuromuscul Disord, 7(5), 277–283. https://doi.org/10.1016/s0960-8966(97)00052-7
Hauser, M. A., A. Amalfitano, R. Kumar-Singh, S. D. Hauschka, and J. S. Chamberlain. “Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy.Neuromuscul Disord 7, no. 5 (July 1997): 277–83. https://doi.org/10.1016/s0960-8966(97)00052-7.
Hauser MA, Amalfitano A, Kumar-Singh R, Hauschka SD, Chamberlain JS. Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy. Neuromuscul Disord. 1997 Jul;7(5):277–83.
Hauser, M. A., et al. “Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy.Neuromuscul Disord, vol. 7, no. 5, July 1997, pp. 277–83. Pubmed, doi:10.1016/s0960-8966(97)00052-7.
Hauser MA, Amalfitano A, Kumar-Singh R, Hauschka SD, Chamberlain JS. Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy. Neuromuscul Disord. 1997 Jul;7(5):277–283.
Journal cover image

Published In

Neuromuscul Disord

DOI

ISSN

0960-8966

Publication Date

July 1997

Volume

7

Issue

5

Start / End Page

277 / 283

Location

England

Related Subject Headings

  • Virus Replication
  • Neurology & Neurosurgery
  • Mutagenesis, Insertional
  • Muscular Dystrophy, Animal
  • Muscles
  • Mice, Transgenic
  • Mice, Inbred mdx
  • Mice
  • Genetic Vectors
  • Genetic Therapy