Open AccessProblems and solutions in myoblast transfer therapy
Author(s) -
Smythe Gayle M.,
Hodgetts Stuart I.,
Grounds Miranda D.
Publication year - 2001
Publication title -
journal of cellular and molecular medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.44
H-Index - 130
eISSN - 1582-4934
pISSN - 1582-1838
DOI - 10.1111/j.1582-4934.2001.tb00136.x
Subject(s) - dystrophin , duchenne muscular dystrophy , muscular dystrophy , wasting , myocyte , genetic enhancement , population , itga7 , neuromuscular disease , biology , skeletal muscle , gene , bioinformatics , medicine , genetics , microbiology and biotechnology , disease , endocrinology , environmental health
Duchenne muscular dystrophy is a severe X‐linked neuromuscular disease that affects approximately 1/3500 live male births in every human population, and is caused by a mutation in the gene that encodes the muscle protein dystrophin. The characterization and cloning of the dystrophin gene in 1987 was a major breakthrough and it was considered that simple replacement of the dystrophin gene would ameliorate the severe and progressive skeletal muscle wasting characteristic of Duchenne muscular dystrophy. After 20 years, attempts at replacing the dystrophin gene either experimentally or clinically have met with little success, but there have been many significant advances in understanding the factors that limit the delivery of a normal dystrophin gene into dystrophic host muscle. This review addresses the host immune response and donor myoblast changes underlying some of the major problems associated with myoblast‐mediated dystrophin replacement, presents potential solutions, and outlines other novel therapeutic approaches.