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JACIII Vol.16 No.4 pp. 521-526
doi: 10.20965/jaciii.2012.p0521
(2012)

Paper:

New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy

Yoshitsugu Aoki, Tetsuya Nagata, and Shin’ichi Takeda

Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), 4-1-1 Ogawa-higashi, Kodaira, Tokyo 187-8502, Japan

Received:
January 25, 2012
Accepted:
April 7, 2012
Published:
June 20, 2012
Keywords:
duchenne muscular dystrophy, exon skipping, antisense therapeutics
Abstract

Duchenne Muscular Dystrophy (DMD) is a lethalmuscle disorder characterized by mutations in the DMD gene. These mutations primarily disrupt the reading frame, resulting in the absence of functional dystrophin protein. Exon skipping, which involves the use of antisense oligonucleotides is a promising therapeutic approach for DMD, and clinical trials on exon skipping are currently underway in DMD patients. Recently, stable and less-toxic antisense oligonucleotides with higher efficacy have been developed in mouse and dog models of DMD. This review highlights a new approach for antisense oligonucleotide-based therapeutics for DMD, particularly for exon skipping-based methods.

Cite this article as:
Yoshitsugu Aoki, Tetsuya Nagata, and Shin’ichi Takeda, “New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy,” J. Adv. Comput. Intell. Intell. Inform., Vol.16, No.4, pp. 521-526, 2012.
Data files:
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