Antisense oligonucleotide mediated therapy of spinal muscular atrophy

Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality. SMA results from deletions or mutations of survival motor neuron 1 (SMN1), an essential gene. SMN2, a nearly identical copy, can compensate for SMN1 loss if SMN2 exon 7 skipping is prevented. Among the many cis-elements...

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Bibliographic Details
Main Authors:	Sivanesan, Senthilkumar, Howell, Matthew D., DiDonato, Christine J., Singh, Ravindra N.
Format:	Artigo
Language:	Inglês
Published:	2013
Subjects:	Article
Online Access:	https://ncbi.nlm.nih.gov/pmc/articles/PMC3832262/ https://ncbi.nlm.nih.gov/pubmed/24265944 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2478/s13380-013-0109-2
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Antisense oligonucleotide mediated therapy of spinal muscular atrophy

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