(Q40480552)

20 September 2017

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

1 reference

20 September 2017

spinal muscular atrophy

1 reference

Mimoun Azzouz

1

1 reference

20 September 2017

Thanh Le

2

1 reference

20 September 2017

G Scott Ralph

3

1 reference

20 September 2017

Lucy Walmsley

4

1 reference

20 September 2017

Umrao R Monani

5

1 reference

20 September 2017

Debbie C P Lee

6

1 reference

20 September 2017

Fraser Wilkes

7

1 reference

20 September 2017

Kyriacos A Mitrophanous

8

1 reference

20 September 2017

Susan M Kingsman

9

1 reference

20 September 2017

Arthur H M Burghes

10

1 reference

20 September 2017

Nicholas D Mazarakis

11

1 reference

20 September 2017

language of work or name

English

0 references

publication date

1 December 2004

1 reference

Journal of Clinical Investigation

20 September 2017

1 reference

20 September 2017

114

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20 September 2017

12

1 reference

20 September 2017

1726-1731

1 reference

Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high-energy diet in a transgenic mouse model

20 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Recombinant human insulin-like growth factor I (rhIGF-I) for amyotrophic lateral sclerosis/motor neuron disease.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Lentiviral vectors for the treatment of neurodegenerative diseases.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Molecular mechanisms in spinal muscular atrophy: models and perspectives

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Nuclear targeting defect of SMN lacking the C-terminus in a mouse model of spinal muscular atrophy

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Spinal muscular atrophy: molecular pathophysiology

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Expression of the SMN gene, the spinal muscular atrophy determining gene, in the mammalian central nervous system

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

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

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

The survival motor neuron protein in spinal muscular atrophy

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

A novel nuclear structure containing the survival of motor neurons protein

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Identification and characterization of a spinal muscular atrophy-determining gene

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Design, production, safety, evaluation, and clinical applications of nonprimate lentiviral vectors

22 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Stable gene transfer to the nervous system using a non-primate lentiviral vector

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Correlation between severity and SMN protein level in spinal muscular atrophy

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

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

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

The genetic component in child mortality

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Wild-Type Nonneuronal Cells Extend Survival of SOD1 Mutant Motor Neurons in ALS Mice

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery

20 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=535071

International SMA consortium meeting. (26-28 June 1992, Bonn, Germany)

20 August 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

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

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

Transduction Patterns of Pseudotyped Lentiviral Vectors in the Nervous System

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

SMN protein analysis in fibroblast, amniocyte and CVS cultures from spinal muscular atrophy patients and its relevance for diagnosis

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

Estimation of nuclear population from microtome sections

1 reference

https://pubmed.ncbi.nlm.nih.gov/15599397

12 December 2020

Identifiers

DOI

10.1172/JCI22922

1 reference

20 September 2017

1 reference

20 September 2017

PubMed publication ID

15599397

1 reference