Correction of RNA Splicing with Antisense Oligonucleotides as a Therapeutic Strategy for a Neurodegenerative Disease

Hua, Yimin, Sahashi, Kentaro, Rigo, Frank, Hung, Gene, Bennett, C. Frank, Krainer, AdrianR (2013) Correction of RNA Splicing with Antisense Oligonucleotides as a Therapeutic Strategy for a Neurodegenerative Disease. In: Chembiomolecular Science. Springer Japan, pp. 301-313. ISBN 978-4-431-54037-3

URL: http://link.springer.com/chapter/10.1007%2F978-4-4...
DOI: 10.1007/978-4-431-54038-0_30

Abstract

Spinal muscular atrophy (SMA) is a severe genetic disease inherited in autosomal recessive fashion. It is the leading genetic cause of infant mortality. SMA is a neuromuscular disease, characterized by progressive degeneration and loss of α-motor neurons in the anterior horn of the spinal cord, which in turn leads to muscle weakness and atrophy, resulting in gradual paralysis. SMA is classified into four types on the basis of severity and time of onset: childhood-onset SMA ranges from type I, which is the most severe, to type III, which is considerably milder, with type II having intermediate severity [1–4]; adult-onset SMA is classified as type IV. There is no effective therapy for SMA.

Item Type: Book Section
Subjects: bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > antisense
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > oligonucleotide
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > RNA splicing
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > protein structure, function, modification > protein types > splicing factor
CSHL Authors:
Communities: CSHL labs > Krainer lab
Depositing User: Matt Covey
Date: 2013
Date Deposited: 11 Mar 2014 21:04
Last Modified: 11 Mar 2014 21:04
URI: http://repository.cshl.edu/id/eprint/29587

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