Antisense-based therapy for the treatment of spinal muscular atrophy

Rigo, F., Hua, Y. M., Krainer, A. R., Bennett, C. F. (October 2012) Antisense-based therapy for the treatment of spinal muscular atrophy. Journal of Cell Biology, 199 (1). pp. 21-25. ISSN 0021-9525

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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.

Item Type:	Paper
Uncontrolled Keywords:	exonic splicing enhancer survival-motor-neuron mouse model cardiac defects oligonucleotide smn2 disease mechanisms delivery therapeutics
Subjects:	diseases & disorders > congenital hereditary genetic diseases bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification diseases & disorders bioinformatics > genomics and proteomics > genetics & nucleic acid processing bioinformatics > genomics and proteomics bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > antisense diseases & disorders > congenital hereditary genetic diseases > spinal muscular atrophy
CSHL Authors:	Hua, Yimin Krainer, Adrian R.
Communities:	CSHL labs > Krainer lab CSHL Cancer Center Program > Gene Regulation and Cell Proliferation
Depositing User:	Matt Covey
Date:	1 October 2012
Date Deposited:	30 Jan 2013 14:39
Last Modified:	13 Oct 2015 16:15
PMCID:	PMC3461520
Related URLs:	Publisher
URI:	https://repository.cshl.edu/id/eprint/27029

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