(Q45865368)

17 December 2017

title

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice (English)

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17 December 2017

author

Sofia Benkhelifa-Ziyyat

object named as

Sofia Benkhelifa-Ziyyat

4

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Philippe Ravassard

object named as

Philippe Ravassard

6

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Nicolas Chatauret

3

object named as

Nicolas Chatauret

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17 December 2017

author name string

Elisa Dominguez

1

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17 December 2017

Thibaut Marais

2

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17 December 2017

Sandra Duque

5

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17 December 2017

Romain Carcenac

7

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17 December 2017

Stéphanie Astord

8

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17 December 2017

Aurélie Pereira de Moura

9

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17 December 2017

Thomas Voit

10

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17 December 2017

Martine Barkats

11

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17 December 2017

publication date

30 November 2010

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17 December 2017

published in

Human Molecular Genetics

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17 December 2017

volume

20

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17 December 2017

issue

4

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17 December 2017

page(s)

681-693

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17 December 2017

The genetic component in child mortality

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Identification and characterization of a spinal muscular atrophy-determining gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Self-complementary AAV vectors; advances and applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Multiple effects of codon usage optimization on expression and immunogenicity of DNA candidate vaccines encoding the human immunodeficiency virus type 1 Gag protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Codon optimization improves heterologous expression of a Schistosoma mansoni cDNA in HEK293 cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Translation efficiency in humans: tissue specificity, global optimization and differences between developmental stages

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Introns increase transcriptional efficiency in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Heterologous introns can enhance expression of transgenes in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Continuous delivery of human and mouse erythropoietin in mice by genetically engineered polymer encapsulated myoblasts

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Recombinant AAV-mediated gene delivery to the central nervous system.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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/10.1093%2FHMG%2FDDQ514

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

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Rho-kinase inactivation prolongs survival of an intermediate SMA mouse model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Deletion of murine SMN exon 7 directed to skeletal muscle leads to severe muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Refined characterization of the expression and stability of the SMN gene products.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

A Drosophila melanogaster model of spinal muscular atrophy reveals a function for SMN in striated muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Multiprotein complexes of the survival of motor neuron protein SMN with Gemins traffic to neuronal processes and growth cones of motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

The spinal muscular atrophy gene product regulates neurite outgrowth: importance of the C terminus.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Axonal-SMN (a-SMN), a protein isoform of the survival motor neuron gene, is specifically involved in axonogenesis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Association of plastin 3 expression with disease severity in spinal muscular atrophy only in postpubertal females.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

A mouse model for spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Vascular perfusion abnormalities in infants with spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Digital necroses and vascular thrombosis in severe spinal muscular atrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Androgen receptor YAC transgenic mice recapitulate SBMA motor neuronopathy and implicate VEGF164 in the motor neuron degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

VEGF is a modifier of amyotrophic lateral sclerosis in mice and humans and protects motoneurons against ischemic death

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Spinal muscular atrophy, Dandy-Walker complex, and cataracts in two siblings: a new entity?

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Cardiovascular disease, vascular risk factors and the incidence of cataract and cataract surgery: the Blue Mountains Eye Study

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Neuroblastoma x spinal cord (NSC) hybrid cell lines resemble developing motor neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

Relax, a novel rat bHLH transcriptional regulator transiently expressed in the ventricular proliferating zone of the developing central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDQ514

21 January 2018

Identifiers

DOI

10.1093/HMG/DDQ514

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17 December 2017

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17 December 2017