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Article

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2016

Osman Erkan Y, Washington Charles W, Kaifer Kevin A, Mazzasette Chiara, Patitucci Teresa N, Florea Kyra M, Simon Madeline E, Ko Chien-Ping, Ebert Allison D, Lorson Christian L

Abstract excerpt

Loss of Survival Motor Neuron-1 (SMN1) causes Spinal Muscular Atrophy, a devastating neurodegenerative disease. SMN2 is a nearly identical copy gene; however SMN2 cannot prevent disease development in the absence of SMN1 since the majority of SMN2-derived transcripts are alternatively spliced, encoding a truncated, unstable protein lacking exon 7. Nevertheless, SMN2 retains the ability to produce low levels of...

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Source

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Identifiers

PMID: 27401142
DOI: 10.1038/mt.2016.145
PMCID: PMC5113110

Genes

SMN2
SMN1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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