Gene Therapy for Duchenne muscular dystrophy

Expert Opinion on Orphan Drugs
Julian N Ramos, Jeffrey S Chamberlain

Abstract

Duchenne muscular dystrophy (DMD) is a relatively common inherited disorder caused by defective expression of the protein dystrophin. The most direct approach to treating this disease would be to restore dystrophin production in muscle. Recent progress has greatly increased the prospects for successful gene therapy of DMD, and here we summarize the most promising developments. Gene transfer using vectors derived from adeno-associated virus (AAV) has emerged as a promising method to restore dystrophin production in muscles bodywide, and represents a treatment option applicable to all DMD patients. Using information gleaned from PubMed searches of the literature, attendance at scientific conferences and results from our own lab, we provide an overview of the potential for gene therapy of DMD using AAV vectors including a summary of promising developments and issues that need to be resolved prior to large-scale therapeutic implementation. Of the many approaches being pursued to treat DMD and BMD, gene therapy based on AAV-mediated delivery of microdystrophin is the most direct and promising method to treat the cause of the disorder. The major challenges to this approach are ensuring that microdystrophin can be delivered safely and...Continue Reading

Citations

Apr 7, 2016·Clinical Proteomics·Yetrib HathoutEric P Hoffman
May 26, 2016·Molecular Therapy. Methods & Clinical Development·Laura Adamson-SmallNathalie Clément
Aug 21, 2016·Human Genetics·Jacqueline N Robinson-Hamm, Charles A Gersbach
Aug 30, 2016·The Journal of Physiology·Nicholas P WhiteheadStanley C Froehner
Dec 20, 2016·Scientific Reports·Nobuhiro MaruyamaYo-Ichi Nabeshima
Mar 4, 2017·Circulation Research·Thomas Doetschman, Teodora Georgieva
Sep 7, 2016·F1000Research·Hayder Abdul-RazakGeorge Dickson
Mar 27, 2019·Cell Proliferation·Sara ChiappalupiGuglielmo Sorci
Jun 20, 2019·Human Molecular Genetics·Ambreen A Sayed-ZahidGregory A Cox
Dec 1, 2017·Science Translational Medicine·Leonela AmoasiiEric N Olson
Dec 1, 2017·Science Translational Medicine·Francesco PuzzoFederico Mingozzi
Feb 2, 2018·Postgraduate Medical Journal·Vassili Crispi, Antonios Matsakas
Sep 6, 2018·Proceedings of the National Academy of Sciences of the United States of America·Peggy P HoLawrence Steinman
Feb 9, 2019·Human Genetics·Peter P Nghiem, Joe N Kornegay
Sep 16, 2019·Neuromuscular Disorders : NMD·Jessica F BoehlerCarl A Morris

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