Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy
MA Hauser, A Amalfitano, R Kumar-Singh… - Neuromuscular …, 1997 - Elsevier
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 …
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 …