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

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 g...

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Main Authors:	Hashim, Hasnur Zaman, Che Abdullah, Shahrin Tarmizi, Wan Sulaiman, Wan Aliaa, Fan, Kee Hoo, Basri, Hamidon
Format:	Article
Language:	English English English
Published:	Elsevier 2014
Subjects:	RC0321 Neuroscience. Biological psychiatry. Neuropsychiatry
Online Access:	http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/1/hunting_for_cure.pdf http://irep.iium.edu.my/36244/4/scopus.pdf http://irep.iium.edu.my/36244/5/Hunting_for_a_cure_The_therapeutic_potential_of_gene_therapy_in.pdf

id	iium-36244
recordtype	eprints
spelling	iium-362442014-11-27T08:51:25Z http://irep.iium.edu.my/36244/ Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Fan, Kee Hoo Basri, Hamidon RC0321 Neuroscience. Biological psychiatry. Neuropsychiatry 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. Elsevier 2014 Article PeerReviewed application/pdf en http://irep.iium.edu.my/36244/1/hunting_for_cure.pdf application/pdf en http://irep.iium.edu.my/36244/4/scopus.pdf application/pdf en http://irep.iium.edu.my/36244/5/Hunting_for_a_cure_The_therapeutic_potential_of_gene_therapy_in.pdf 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 http://www.sciencedirect.com/science/article/pii/S101631901400007X# 0.1016/j.tcmj.2014.02.002
repository_type	Digital Repository
institution_category	Local University
institution	International Islamic University Malaysia
building	IIUM Repository
collection	Online Access
language	English English English
topic	RC0321 Neuroscience. Biological psychiatry. Neuropsychiatry
spellingShingle	RC0321 Neuroscience. Biological psychiatry. Neuropsychiatry Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Fan, Kee Hoo Basri, Hamidon Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
description	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.
format	Article
author	Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Fan, Kee Hoo Basri, Hamidon
author_facet	Hashim, Hasnur Zaman Che Abdullah, Shahrin Tarmizi Wan Sulaiman, Wan Aliaa Fan, Kee Hoo Basri, Hamidon
author_sort	Hashim, Hasnur Zaman
title	Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
title_short	Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
title_full	Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
title_fullStr	Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
title_full_unstemmed	Hunting for a cure: the therapeutic potential of gene therapy in Duchenne muscular dystrophy
title_sort	hunting for a cure: the therapeutic potential of gene therapy in duchenne muscular dystrophy
publisher	Elsevier
publishDate	2014
url	http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/ http://irep.iium.edu.my/36244/1/hunting_for_cure.pdf http://irep.iium.edu.my/36244/4/scopus.pdf http://irep.iium.edu.my/36244/5/Hunting_for_a_cure_The_therapeutic_potential_of_gene_therapy_in.pdf
first_indexed	2023-09-18T20:51:53Z
last_indexed	2023-09-18T20:51:53Z
_version_	1777410048631242752

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

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