PMID: 11898504Mar 20, 2002Paper

Progress in gene therapy for Duchenne muscular dystrophy

Current Neurology and Neuroscience Reports
P R Clemens, F J Duncan

Abstract

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.

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Citations

Jun 1, 2005·Hearing Research·Sigrid E MyrdalPeter S Steyger
Jun 2, 2012·Expert Opinion on Biological Therapy·Toshifumi YokotaHanna Kolski
Feb 6, 2003·Cytotherapy·F Rivier, E Gussoni
Oct 12, 2018·Journal of Movement Disorders·Ji-Hyun ChoiBeomseok Jeon
Oct 24, 2002·Current Opinion in Clinical Nutrition and Metabolic Care·Mark A Tarnopolsky, Wim Saris

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