(Q27007782)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

1 reference

29 October 2020

Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA) (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

spinal muscular atrophies

4 March 2020

main subject

1 reference

cell- and tissue-based therapy

3 August 2019

1 reference

Survival of motor neuron 2, centromeric

19 September 2019

1 reference

stem cell transplantation

20 December 2019

1 reference

15 October 2020

1 reference

29 October 2020

spinal muscular atrophy

1 reference

1 reference

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Irene Faravelli

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Nereo Bresolin

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Giacomo P Comi

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Stefania Corti

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

author name string

Chiara Zanetta

1

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Giulietta Riboldi

2

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

Chiara Simone

4

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

language of work or name

1 reference

8 January 2014

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

Journal of Cellular and Molecular Medicine

4 March 2020

1 February 2014

2 references

15 October 2020

29 October 2020

10 references

25 April 2019

25 June 2019

3 August 2019

19 September 2019

28 November 2019

20 December 2019

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

15 October 2020

29 October 2020

18

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

issue

2

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

page(s)

187-196

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

Creative Commons Attribution 3.0 Unported

4 March 2020

copyright license

8 references

25 April 2019

25 June 2019

3 August 2019

19 September 2019

28 November 2019

20 December 2019

15 October 2020

on focus list of Wikimedia project

29 October 2020

copyright status

copyrighted

0 references

ScienceSource

0 references

cites work

Spinal muscular atrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Spinal muscular atrophy: SMN2 pre-mRNA splicing corrected by a U7 snRNA derivative carrying a splicing enhancer sequence

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Correction of SMN2 Pre-mRNA splicing by antisense U7 small nuclear RNAs

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Genetic mapping of chronic childhood-onset spinal muscular atrophy to chromosome 5q11.2-13.3

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Derivation of high purity neuronal progenitors from human embryonic stem cells

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Consensus Statement for Standard of Care in Spinal Muscular Atrophy

21 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Identification and characterization of a spinal muscular atrophy-determining gene

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Directed differentiation of embryonic stem cells into motor neurons

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

An intronic structure enabled by a long-distance interaction serves as a novel target for splicing correction in spinal muscular atrophy

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

A critical smn threshold in mice dictates onset of an intermediate spinal muscular atrophy phenotype associated with a distinct neuromuscular junction pathology.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Targeting RNA to treat neuromuscular disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Human motor neuron progenitor transplantation leads to endogenous neuronal sparing in 3 models of motor neuron loss

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Temporal requirement for high SMN expression in SMA mice

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Systemic administration of PRO051 in Duchenne's muscular dystrophy

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Spinal muscular atrophy: a time for screening

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Self-complementary AAV vectors; advances and applications

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Adeno-associated virus (AAV) vectors in the CNS.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Selective and rapid uptake of adeno-associated virus type 2 in brain

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Transplanted ALDHhiSSClo neural stem cells generate motor neurons and delay disease progression of nmd mice, an animal model of SMARD1.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

Human motor neuron differentiation from human embryonic stem cells

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3930406

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

31 May 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

Correction of disease-associated exon skipping by synthetic exon-specific activators

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

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/10.1111%2FJCMM.12224

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJCMM.12224

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

21 January 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/24400925

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Genetic risk assessment in carrier testing for spinal muscular atrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/24400925

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1111/JCMM.12224

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

1 reference

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json

4 March 2020

PubMed publication ID

24400925

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24400925%20AND%20SRC:MED&resulttype=core&format=json