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Journal article

Splicing therapy for neuromuscular disease.

Abstract:

Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two of the most common inherited neuromuscular diseases in humans. Both conditions are fatal and no clinically available treatments are able to significantly alter disease course in either case. However, by manipulation of pre-mRNA splicing using antisense oligonucleotides, defective transcripts from the DMD gene and from the SMN2 gene in SMA can be modified to once again produce protein and restore function. A large numb...

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Publisher copy:
10.1016/j.mcn.2013.04.005

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
Journal:
Molecular and cellular neurosciences More from this journal
Volume:
56
Pages:
169-185
Publication date:
2013-09-01
DOI:
EISSN:
1095-9327
ISSN:
1044-7431
Language:
English
Keywords:
Pubs id:
pubs:401960
UUID:
uuid:6862beef-f911-473d-943a-10bf8b7dc7f9
Local pid:
pubs:401960
Source identifiers:
401960
Deposit date:
2013-12-13

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