(Q34122981)

31 July 2017

title

Therapy development in spinal muscular atrophy (English)

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31 July 2017

main subject

spinal muscular atrophy

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Michael Sendtner

1

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language of work or name

English

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publication date

1 July 2010

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31 July 2017

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31 July 2017

volume

13

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31 July 2017

issue

7

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31 July 2017

page(s)

795-799

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Amyotrophic lateral sclerosis as a complex genetic disease

31 July 2017

cites work

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FNN.2565

Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy.

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FNN.2565

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FNN.2565

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

21 January 2018

1 reference

12 December 2020

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

1 reference

12 December 2020

An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA).

1 reference

12 December 2020

The neurobiology of childhood spinal muscular atrophy.

1 reference

12 December 2020

Modulating role of RNA structure in alternative splicing of a critical exon in the spinal muscular atrophy genes.

1 reference

12 December 2020

Phase II open label study of valproic acid in spinal muscular atrophy

1 reference

12 December 2020

Neurotrophins: from enthusiastic expectations through sobering experiences to rational therapeutic approaches

1 reference

12 December 2020

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

1 reference

12 December 2020

Induced pluripotent stem cells from a spinal muscular atrophy patient

1 reference

12 December 2020

SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN

1 reference

12 December 2020

A degron created by SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity

1 reference

12 December 2020

Progress in therapeutic antisense applications for neuromuscular disorders

1 reference

12 December 2020

A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy

1 reference

12 December 2020

Immunity to adeno-associated virus vectors in animals and humans: a continued challenge

1 reference

12 December 2020

Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy

1 reference

12 December 2020

Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding.

1 reference

12 December 2020

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

1 reference

12 December 2020

The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy

1 reference

12 December 2020

Valproic acid blocks excitability in SMA type I mouse motor neurons

1 reference

12 December 2020

Reduced survival motor neuron (Smn) gene dose in mice leads to motor neuron degeneration: an animal model for spinal muscular atrophy type III

1 reference

12 December 2020

Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice

1 reference

12 December 2020

Splicing therapeutics in SMN2 and APOB.

1 reference

12 December 2020

Establishing a standardized therapeutic testing protocol for spinal muscular atrophy

1 reference

12 December 2020

Histone deacetylase inhibitors

1 reference

12 December 2020

Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

1 reference

12 December 2020

Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6.

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

1 reference

12 December 2020

Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1.

1 reference

12 December 2020

Smn, the spinal muscular atrophy-determining gene product, modulates axon growth and localization of beta-actin mRNA in growth cones of motoneurons

1 reference

12 December 2020

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

1 reference

12 December 2020

VEGF: once regarded as a specific angiogenic factor, now implicated in neuroprotection

1 reference

12 December 2020

Treatment of spinal muscular atrophy by sodium butyrate

1 reference

12 December 2020

Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons

1 reference

12 December 2020

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

12 December 2020

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon

1 reference

12 December 2020

Recent advances in the genetics of amyotrophic lateral sclerosis

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12 December 2020

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

1 reference

12 December 2020

Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice.

1 reference

12 December 2020

Valproic acid increases SMN levels in spinal muscular atrophy patient cells.

1 reference

12 December 2020

Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.

1 reference

12 December 2020

Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos

1 reference

12 December 2020

Specific interaction of Smn, the spinal muscular atrophy determining gene product, with hnRNP-R and gry-rbp/hnRNP-Q: a role for Smn in RNA processing in motor axons?

1 reference

12 December 2020

Antisense applications for biological control

1 reference

12 December 2020

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

1 reference

12 December 2020

Unaffected patients with a homozygous absence of the SMN1 gene

1 reference

12 December 2020

Defective Ca2+ channel clustering in axon terminals disturbs excitability in motoneurons in spinal muscular atrophy

1 reference

12 December 2020

Identifiers

DOI

10.1038/NN.2565

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31 July 2017

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