Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy (Q44329621)

2 December 2017

title

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy (English)

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2 December 2017

main subject

spinal muscular atrophy

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5

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6

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7

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2 December 2017

Richard J Mead

4

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Ke Ning

object named as

Ke Ning

series ordinal

2

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author name string

Chiara F Valori

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1

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2 December 2017

Matthew Wyles

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3

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2 December 2017

language of work or name

English

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publication date

1 June 2010

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Science Translational Medicine

2 December 2017

published in

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2 December 2017

volume

2

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2 December 2017

issue

35

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2 December 2017

page(s)

35ra42

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2 December 2017

cites work

Spinal muscular atrophy.

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Identification and characterization of a spinal muscular atrophy-determining gene

21 January 2018

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

21 January 2018

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

21 January 2018

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Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

21 January 2018

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

21 January 2018

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Gene therapy progress and prospects: recent progress in transgene and RNAi expression cassettes

21 January 2018

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

21 January 2018

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Codon bias and heterologous protein expression

21 January 2018

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The survival motor neuron protein in spinal muscular atrophy

21 January 2018

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Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

21 January 2018

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Identification of a battery of tests for drug candidate evaluation in the SMNDelta7 neonate model of spinal muscular atrophy.

21 January 2018

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Abnormal motor phenotype in the SMNDelta7 mouse model of spinal muscular atrophy

21 January 2018

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A mouse model for spinal muscular atrophy

21 January 2018

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Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition

21 January 2018

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Vascular perfusion abnormalities in infants with spinal muscular atrophy

21 January 2018

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Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

21 January 2018

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Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

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

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3000830

10.1126/SCITRANSLMED.3000830

21 January 2018

Identifiers

DOI

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2 December 2017

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