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 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.
Clemens
P R
PR
Department of Neurology, University of Pittsburgh, Room S-515 Biomedical Science Tower South, Pittsburgh, PA 15213, USA. pclemens+@pitt.edu
Duncan
F J
FJ
D000900:Anti-Bacterial Agents / Q000627:therapeutic use; D003598:Cytoskeletal Proteins / Q000627:therapeutic use; D015316:Genetic Therapy; D005822:Genetic Vectors; D005839:Gentamicins / Q000627:therapeutic use; D006801:Humans; D008565:Membrane Proteins / Q000627:therapeutic use; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy / Q000235:genetics / Q000628:therapy*; D049411:Utrophin
D000900:Anti-Bacterial Agents; D003598:Cytoskeletal Proteins; D005839:Gentamicins; D008565:Membrane Proteins; D049411:Utrophin