Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy

Hashim, Hasnur Zaman and Che Abdullah, Shahrin Tarmizi and Wan Sulaiman, Wan Aliaa and Fan, Kee Hoo and Basri, Hamidon (2014) Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy. Tzu Chi Medical Journal, 26 (1). pp. 5-9. ISSN 1016-3190

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Abstract

Duchenne muscular dystrophy (DMD) is an incurable disease and the search for a cure is a challenging journey. However, with recent encouraging progress, we are seeing a light at the end of a long tunnel. This review focuses on several main strategies in gene therapy, including truncated dystrophin gene transfer via viral vectors, antisense mediated exon skipping to restore the reading frame, and read-through of translation stop codons. An exon skipping agent, eteplirsen, and a termination codon read drug, ataluren, are currently the most promising therapies. With better understanding of the molecular mechanism, gene therapy has improved with regard to the key areas of gene stability, safety, and route of delivery. Consequently, it has emerged as an exciting and hopeful means for novel treatment of this devastating disease.

Item Type:	Article (Journal)
Additional Information:	5220/36244
Uncontrolled Keywords:	Cure; Duchenne; Gene therapy; Muscular dystrophy
Subjects:	R Medicine > RC Internal medicine > RC321 Neuroscience. Biological psychiatry. Neuropsychiatry
Kulliyyahs/Centres/Divisions/Institutes (Can select more than one option. Press CONTROL button):	Kulliyyah of Medicine > Department of Internal Medicine
Depositing User:	Dr Hasnur Zaman Hashim
Date Deposited:	03 Apr 2014 15:40
Last Modified:	27 Nov 2014 16:51
URI:	http://irep.iium.edu.my/id/eprint/36244

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