ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.

Volume: 8, Issue: 1, Pages: 1 - 6
Published: Jan 26, 2017
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
Volume
8
Issue
1
Pages
1 - 6
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