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1-3 of 3 results for ISS-N1

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Select article: A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

Retricted access

Research article

First published 2013-03

A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice

Haiyan Zhou, Narinder Janghra, Chalermchai Mitrpant [...]

Human Gene Therapy

Select article: The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Open access

Review article

First published 2020

The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy

Ravindra N Singh, Eric W Ottesen, Natalia N Singh

Neuroscience Insights

Select article: Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Open access

Article commentary

First published 2024

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Eric W Ottesen, Ravindra N Singh

Neuroscience Insights

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