Progress in gene therapy for Duchenne muscular dystrophy

Gene transfer research for Duchenne muscular dystrophy (DMD) has brought the goal of successful treatment of this devastating, inherited disease closer to being a reality. Although gene therapeutic approaches for DMD patients are not yet in clinical use, recent advances using DMD animal models are e...

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Bibliographic Details
Published in:Current neurology and neuroscience reports 2001, Vol.1 (1), p.89-96
Main Authors: Clemens, P R, Duncan, F J
Format: Article
Language:English
Subjects:
Anti-Bacterial Agents - therapeutic use
Cytoskeletal Proteins - therapeutic use
Gene therapy
Genetic Therapy
Genetic Vectors
Gentamicins - therapeutic use
Humans
Membrane Proteins - therapeutic use
Muscular dystrophy
Muscular Dystrophy, Duchenne - drug therapy
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - therapy
Utrophin
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Summary:Gene transfer research for Duchenne muscular dystrophy (DMD) has brought the goal of successful treatment of this devastating, inherited disease closer to being a reality. Although gene therapeutic approaches for DMD patients are not yet in clinical use, recent advances using DMD animal models are encouraging. Progress in vector design, such as high-capacity adenoviral vectors, targeted adenoviral vectors, and heterodimerization of DNA delivered by adeno-associated virus (AAV) vectors have advanced the field considerably. The recent studies into the pharmacologic-induced read-through of stop codons, the increased study of utrophin and its upregulation, and the introduction of point mutation correction using chimeric oligonucleotides have expanded the field, providing new avenues of inquiry.
ISSN:1528-4042
1534-6293
DOI:10.1007/s11910-001-0080-0