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Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice

Neurobiology of disease, 2021-11, Vol.159, p.105488-105488, Article 105488

2021

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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

Human molecular genetics, 2012-04, Vol.21 (7), p.1625-1638

2012

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What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective

International journal of molecular sciences, 2021-08, Vol.22 (16), p.8494

2021

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Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Human molecular genetics, 2010-07, Vol.19 (20), p.3895-3905

2010

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A Role for SMN Exon 7 Splicing in the Selective Vulnerability of Motor Neurons in Spinal Muscular Atrophy

Molecular and Cellular Biology, 2012-01, Vol.32 (1), p.126-138

2012

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The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy

Neurobiology of disease, 2016-03, Vol.87, p.116-123

2016

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Mild SMN missense alleles are only functional in the presence of SMN2 in mammals

Human molecular genetics, 2018-10, Vol.27 (19), p.3404-3416

2018

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Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

PloS one, 2016-12, Vol.11 (12), p.e0167077-e0167077

2016

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Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

Nature medicine, 2021-10, Vol.27 (10), p.1701-1711

2021

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Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse

PloS one, 2015, Vol.10 (7), p.e0132364-e0132364

2015

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Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Nature biotechnology, 2010-03, Vol.28 (3), p.271-274

2010

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Conditional deletion of SMN in cell culture identifies functional SMN alleles

Human molecular genetics, 2020-11, Vol.29 (21), p.3477-3492

2020

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Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotype

Human genetics, 2019-03, Vol.138 (3), p.241-256

2019

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Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Nature biotechnology, 2022-11, Vol.40 (11), p.1692-1692

2022

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SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA

Human molecular genetics, 2015-10, Vol.24 (19), p.5524-5541

2015

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Temporal requirement for high SMN expression in SMA mice

Human molecular genetics, 2011-09, Vol.20 (18), p.3578-3591

2011

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SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMN Delta 7 mouse model of SMA

Human molecular genetics, 2015-10, Vol.24 (19), p.5524-5541

2015

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Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice

Human molecular genetics, 2020-11, Vol.29 (21), p.3493-3503

2020

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Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA

Human molecular genetics, 2015-11, Vol.24 (21), p.6160-6173

2015

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Embryonic motor axon development in the severe SMA mouse

Human molecular genetics, 2008-09, Vol.17 (18), p.2900-2909

2008

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Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

Human molecular genetics, 2008-04, Vol.17 (8), p.1063-1075

2008

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Mild SMN missense alleles are only functional in the presence of SMN2 in mammals

Human molecular genetics, 2018-10, Vol.27 (19), p.3404-3416

2018

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SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice

Human molecular genetics, 2015-07, Vol.24 (13), p.3847-3860

2015

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Dual SMN inducing therapies can rescue survival and motor unit function in symptomatic ∆7SMA mice

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective

Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

A Role for SMN Exon 7 Splicing in the Selective Vulnerability of Motor Neurons in Spinal Muscular Atrophy

The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy

Mild SMN missense alleles are only functional in the presence of SMN2 in mammals

Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

Natural history of infantile‐onset spinal muscular atrophy

Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Conditional deletion of SMN in cell culture identifies functional SMN alleles

Complete sequencing of the SMN2 gene in SMA patients detects SMN gene deletion junctions and variants in SMN2 that modify the SMA phenotype

Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA

Temporal requirement for high SMN expression in SMA mice

SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMN Delta 7 mouse model of SMA

Intragenic complementation of amino and carboxy terminal SMN missense mutations can rescue Smn null mice

Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA

Embryonic motor axon development in the severe SMA mouse

Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect

Mild SMN missense alleles are only functional in the presence of SMN2 in mammals

SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

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