Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Abstract

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by mutations in the SMN1 gene that result in a deficiency of SMN protein. One approach to treat SMA is to use antisense oligonucleotides (ASOs) to redirect the splicing of a paralogous gene, SMN2, to boost production of functional SMN. Injection of a 2'-O-2-methoxyethyl… (More)
DOI: 10.1126/scitranslmed.3001777

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Cite this paper

@article{Passini2011AntisenseOD, title={Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.}, author={Marco A. Passini and Jie Bu and Amy M Richards and Cathrine Kinnecom and Sergio Pablo Sardi and Lisa M. Stanek and Yimin Hua and Frank Rigo and John E. Matson and Gene H Hung and Edward M. Kaye and Lamya S. Shihabuddin and Adrian R. Krainer and C. F. Bennett and Seng H. Cheng}, journal={Science translational medicine}, year={2011}, volume={3 72}, pages={72ra18} }