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Developments in gene therapy for muscular dystrophy
Author(s) -
HartiganO'Connor Dennis,
Chamberlain Jeffrey S.
Publication year - 2000
Publication title -
microscopy research and technique
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.536
H-Index - 118
eISSN - 1097-0029
pISSN - 1059-910X
DOI - 10.1002/(sici)1097-0029(20000201/15)48:3/4<223::aid-jemt10>3.0.co;2-l
Subject(s) - muscular dystrophy , genetic enhancement , gene , computational biology , biology , genetics , medicine , bioinformatics
Gene therapy for muscular dystrophy (MD) presents significant challenges, including the large amount of muscle tissue in the body, the large size of many genes defective in different muscular dystrophies, and the possibility of a host immune response against the therapeutic gene. Overcoming these challenges requires the development and delivery of suitable gene transfer vectors. Encouraging progress has been made in modifying adenovirus (Ad) vectors to reduce immune response and increase capacity. Recently developed gutted Ad vectors can deliver full‐length dystrophin cDNA expression vectors to muscle tissue. Using muscle‐specific promoters to drive dystrophin expression, a strong immune response has not been observed in mdx mice. Adeno‐associated virus (AAV) vectors can deliver small genes to muscle without provocation of a significant immune response, which should allow long‐term expression of several MD genes. AAV vectors have also been used to deliver sarcoglycan genes to entire muscle groups. These advances and others reviewed here suggest that barriers to gene therapy for MD are surmountable. Microsc. Res. Tech. 48:223–238, 2000. © 2000 Wiley‐Liss, Inc.