The future of Duchenne muscular dystrophy gene therapy: shrinking the dystrophin gene.
Duchenne muscular dystrophy is a debilitating muscle-wasting disease caused by mutations in the dystrophin gene - one of the largest genes identified thus far - and which ultimately results in premature death. With no current treatment available, the hopes of many sufferers lie in the establishment of an effective gene therapy. The adeno-associated virus is now emerging as a premium gene transfer vector eliciting minimal immune response from the host and allowing for long-term gene expression. It is the scope of this review to examine the recent efforts that have been made to develop ultra-truncated versions of the dystrophin gene that retain functionality, yet can still be cloned into recombinant adeno-associated viral vectors and other low-capacity vector systems.
Roberts
Michael
M
Institute of Biological Research and Biotechnology, National Hellenic Research Foundation, Athens, Greece.
Dickson
George
G
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D016189:Dystrophin / Q000235:genetics*; D005544:Forecasting; D015316:Genetic Therapy / Q000639:trends; D005822:Genetic Vectors; D006801:Humans; D006824:Hybridization, Genetic; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000628:therapy*; D012190:Retroviridae / Q000235:genetics; D017384:Sequence Deletion; D013049:Spectrin / Q000235:genetics
D016189:Dystrophin; D013049:Spectrin