(Q34695728)

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Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy (English)

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

spinal muscular atrophy

1 reference

based on heuristic

inferred from title

muscular atrophy

1 reference

based on heuristic

inferred from title

author name string

Chalermchai Mitrpant

1

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Paul Porensky

2

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Haiyan Zhou

3

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Loren Price

4

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Francesco Muntoni

5

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Sue Fletcher

6

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Steve D Wilton

7

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

Arthur H M Burghes

8

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

language of work or name

0 references

publication date

22 April 2013

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

8

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

4

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

e62114

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

copyright license

Creative Commons Attribution 4.0 International

22 April 2013

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Splicing of the Survival Motor Neuron genes and implications for treatment of SMA

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Vascular perfusion abnormalities in infants with spinal muscular atrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Rational design of antisense oligomers to induce dystrophin exon skipping.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Regulation of SMN protein stability

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

The influence of antisense oligonucleotide length on dystrophin exon skipping.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Identification and characterization of a spinal muscular atrophy-determining gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Survival motor neuron gene transcript analysis in muscles from spinal muscular atrophy patients.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

The genetic component in child mortality

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3632594

7 July 2018

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0062114

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0062114

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0062114

21 January 2018

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0062114

21 January 2018

Single section Western blot: Improving the molecular diagnosis of the muscular dystrophies

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0062114

21 January 2018

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/23630626

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

SMN oligomerization defect correlates with spinal muscular atrophy severity

1 reference

https://pubmed.ncbi.nlm.nih.gov/23630626

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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/23630626

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1371/JOURNAL.PONE.0062114

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

2013PLoSO...862114M

0 references

release_rj4yfiw3bvchpgrevqzalgzyii

1 reference

https://api.fatcat.wiki/v0/release/rj4yfiw3bvchpgrevqzalgzyii

24 November 2022

based on heuristic

mapped directly with Wikidata item

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

4 August 2017

ResearchGate publication ID

0 references

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