(Q33598293)

28 July 2017

Exon exchange approach to repair Duchenne dystrophin transcripts (English)

1 reference

28 July 2017

Cécile Peccate

2

0 references

Stéphanie Lorain

object named as

Stéphanie Lorain

series ordinal

1

0 references

author name string

Maëva Le Hir

series ordinal

3

1 reference

PubMed ID

20531943

28 July 2017

Luis Garcia

series ordinal

4

1 reference

PubMed ID

20531943

28 July 2017

language of work or name

English

0 references

publication date

28 May 2010

1 reference

28 July 2017

1 reference

28 July 2017

5

1 reference

28 July 2017

5

1 reference

28 July 2017

e10894

1 reference

Creative Commons Attribution 4.0 International

28 July 2017

start time

28 May 2010

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

0 references

cites work

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Development of a single vector system that enhances trans-splicing of SMN2 transcripts

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Identification of an intronic splicing enhancer essential for the inclusion of FGFR2 exon IIIc

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

microRNAs: a new emerging class of players for disease diagnostics and gene therapy

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Using 5'-PTMs to repair mutant beta-globin transcripts

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Gene therapy progress and prospects: reprograming gene expression by trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

RNA repair using spliceosome-mediated RNA trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

5' exon replacement and repair by spliceosome-mediated RNA trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Characterization of an intron splice enhancer that regulates alternative splicing of human GH pre-mRNA.

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice

21 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2878348

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 June 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

Spliceosome-mediated RNA trans-splicing as a tool for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

Spliceosome-mediated RNA trans-splicing with recombinant adeno-associated virus partially restores cystic fibrosis transmembrane conductance regulator function to polarized human cystic fibrosis airway epithelial cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0010894

The molecular basis of muscular dystrophy in the mdx mouse: a point mutation

21 January 2018

1 reference

12 December 2020

Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene

1 reference

12 December 2020

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

10.1371/JOURNAL.PONE.0010894

Identifiers

DOI

1 reference

28 July 2017

0 references

1 reference

28 July 2017

1 reference

PubMed ID

20531943