Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy (Q34168139)

31 July 2017

title

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

1 reference

31 July 2017

main subject

spinal muscular atrophy

1 reference

based on heuristic

inferred from title

author

Adrian Robert Krainer

13

object named as

Adrian R Krainer

1 reference

31 July 2017

Marco A Passini

1

1 reference

31 July 2017

Jie Bu

2

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31 July 2017

Amy M Richards

3

1 reference

31 July 2017

Cathrine Kinnecom

4

1 reference

31 July 2017

S Pablo Sardi

5

1 reference

31 July 2017

Lisa M Stanek

6

1 reference

31 July 2017

Yimin Hua

7

1 reference

31 July 2017

Frank Rigo

8

1 reference

31 July 2017

John Matson

9

1 reference

31 July 2017

Gene Hung

10

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31 July 2017

Edward M Kaye

11

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31 July 2017

Lamya S Shihabuddin

12

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31 July 2017

C Frank Bennett

14

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31 July 2017

Seng H Cheng

15

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31 July 2017

language of work or name

English

0 references

publication date

1 March 2011

1 reference

Science Translational Medicine

31 July 2017

published in

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31 July 2017

volume

3

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31 July 2017

issue

72

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31 July 2017

page(s)

72ra18

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

31 July 2017

cites work

1 reference

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5 July 2018

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5 July 2018

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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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

5 July 2018

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5 July 2018

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5 July 2018

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A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy

5 July 2018

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5 July 2018

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Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy

5 July 2018

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Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy

5 July 2018

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Congenital heart disease is a feature of severe infantile spinal muscular atrophy

5 July 2018

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

5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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Medical considerations of long-term survival of Werdnig-Hoffmann disease

5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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5 July 2018

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Quantitation of phosphorothioate oligonucleotides in human plasma

5 July 2018

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Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain

5 July 2018

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Identification and characterization of a spinal muscular atrophy-determining gene

5 July 2018

1 reference

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Homozygous SMN1 deletions in unaffected family members and modification of the phenotype by SMN2.

5 July 2018

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

21 January 2018

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001777

21 January 2018

Spinal muscular atrophy

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https://api.crossref.org/works/10.1126%2FSCITRANSLMED.3001777

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

21 January 2018

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Autonomic dysfunction in cases of spinal muscular atrophy type 1 with long survival

21 January 2018

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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

21 January 2018

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PubMed

https://pubmed.ncbi.nlm.nih.gov/21368223

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.3001777

1 reference

31 July 2017

1 reference

31 July 2017

1 reference