Gene therapy for muscular dystrophies: current status and future prospects

BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
S Takeda, Y Miyagoe-Suzuki

Abstract

Since the identification in 1987 of the gene for Duchenne muscular dystrophy (DMD), research on the molecular pathogenesis of muscular dystrophy has progressed extensively. In particular, discovery of the DMD gene product, dystrophin, led to the identification of dystrophin-associated proteins and, subsequently, the recognition of other types of muscular dystrophy caused by the defects in each of the sarcoglycan genes. On the other hand, effective therapy for DMD has not yet been established. Some of the viral vectors, such as adeno-associated virus vectors or lentiviral vector, have been proven to enable the long-term expression of the exogenous gene without overt host immune reactions. However, dystrophin cDNAs are too large (14kb) to be accommodated in these viral vectors. To solve this problem, we and other research groups succeeded in truncating full-length dystrophin cDNA to small dystrophin cDNA (4 to 5kb), the products of which protect dystrophin-deficient mdx muscle from contraction-induced membrane damage when introduced by viral vectors or as a transgene into mdx mice. The usefulness of these truncated dystrophin cDNAs should be confirmed using other animal models such as dystrophic dogs. To develop successful treatm...Continue Reading

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Citations

Jun 11, 2002·Biochemical and Biophysical Research Communications·Miki SakamotoShin'ichi Takeda
Nov 6, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·En KimuraJeffrey S Chamberlain
Dec 22, 2004·Human Gene Therapy·Norma B RomeroMichel Fardeau
Sep 6, 2003·The Journal of Pharmacy and Pharmacology·Thomas P JohnstonAshim K Mitra
Sep 29, 2004·Molecular Therapy : the Journal of the American Society of Gene Therapy·Simon P QuennevilleJacques P Tremblay
Jul 5, 2005·The Journal of Histochemistry and Cytochemistry : Official Journal of the Histochemistry Society·Kristina R MuskiewiczEmanuela Gussoni
Sep 6, 2003·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Eva Engvall, Ulla M Wewer

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