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PMID: 23027901 Published · ppublish English Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Antisense-based therapy for the treatment of spinal muscular atrophy.

The Journal of cell biology ·Vol. 199 ·No. 1 ·2012-10-01 ·Pages 21-5

Rigo F, Hua Y, Krainer AR, Bennett CF

Abstract

One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.

MeSH Terms
Animals Humans Mice Mice, Transgenic Muscular Atrophy, Spinal/drug therapy,genetics Oligonucleotides, Antisense/pharmacology RNA, Messenger/drug effects,genetics Survival of Motor Neuron 1 Protein/antagonists & inhibitors,genetics Survival of Motor Neuron 2 Protein/antagonists & inhibitors,genetics
Chemicals
Oligonucleotides, Antisense RNA, Messenger SMN1 protein, human SMN2 protein, human Survival of Motor Neuron 1 Protein Survival of Motor Neuron 2 Protein
Authors & Affiliations
4 authors, click to expand affiliations / ORCID
Rigo Frank
Isis Pharmaceuticals, Carlsbad, CA 92010, USA.
Hua Yimin
Krainer Adrian R
Bennett C Frank
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36 references, click to expand
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Article Info
Journal
The Journal of cell biology
Abbr.
J Cell Biol
ISSN
1540-8140
DOI
10.1083/jcb.201207087
Published
2012-10-01
Pages
21-5
Language
English
Region
United States
NLM ID
0375356
PMCID
PMC3461520
Subset
IM
Grants
NIGMS NIH HHS · R37 GM042699 · United States
NIGMS NIH HHS · R37 GM42699-22 · United States
External Links
PubMed source DOI: 10.1083/jcb.201207087 PMC Full Text View journal details
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