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Retricted access

Research article

First published 2019-04

A SMN2 Splicing Modifier Rescues the Disease Phenotypes in an In Vitro Human Spinal Muscular Atrophy Model

Ye Seul Son, Kwangman Choi, Hana Lee [...]

Stem Cells and Development

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Review article

First published 2023-03

An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy

Yueyi Li, Hongyu Zeng, Yuhao Wei [...]

Human Gene Therapy

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Research article

First published 2011-02

Combination of SMN Trans -Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular Atrophy

Monir Shababi, Jacqueline Glascock, Christian L. Lorson

Human Gene Therapy

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Research article

First published 2007-12

An SMA Project Report: Neural Cell-Based Assays Derived from Human Embryonic Stem Cells

Patricia G. Wilson, Jonathan J. Cherry, Shelle Schwamberger [...]

Stem Cells and Development

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Research article

First published 2008-11

A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both In Vitro and In Vivo

Alexa Dickson, Erkan Osman, Christian L. Lorson

Human Gene Therapy

Open access

Review article

First published 2014-08

Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy

Jonathan J. Cherry, Dione T. Kobayashi, Maureen M. Lynes [...]

ASSAY and Drug Development Technologies

Free access

Research article

First published 2016-11-29

Type 0 Spinal Muscular Atrophy: Further Delineation of Prenatal and Postnatal Features in 16 Patients

Sarah Grotto, Jean-Marie Cuisset, Stéphane Marret [...]

Journal of Neuromuscular Diseases

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Brief report

First published 2012-03

Decreasing Disease Severity in Symptomatic,Smn −/−;SMN2 +/+,Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Jacqueline J. Glascock, Erkan Y. Osman, Mary J. Wetz [...]

Human Gene Therapy

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Research article

First published 2005-07

In Vitro Restoration of Functional SMN Protein in Human Trophoblast Cells Affected by Spinal Muscular Atrophy by Small Fragment Homologous Replacement

Federica Sangiuolo, Antonio Filareto, Paola Spitalieri [...]

Human Gene Therapy

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Review article

First published 2013-05

Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy

Paul N. Porensky, Arthur H.M. Burghes

Human Gene Therapy

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