ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.
Abstract
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, cannot compensate for the loss of SMN1 due to predominant skipping of exon 7. While various regulatory elements that modulate SMN2 exon 7 splicing have been proposed, intronic splicing silencer N1 (ISS-N1) has emerged as the most...
Paper Details
Title
ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.
Published Date
Jan 26, 2017
Journal
Volume
8
Issue
1
Pages
1 - 6
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Notes
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