A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence (Q40884119)

24 September 2017

A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence (English)

1 reference

24 September 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Marina Pryadkina

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Nathalie Bourg

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Karine Charton

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Carinne Roudaut

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Matthew L Hirsch

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Isabelle Richard

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

language of work or name

1 reference

25 March 2015

1 reference

Molecular Therapy. Methods & Clinical Development

24 September 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

volume

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

page(s)

15009

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

Full-length dystrophin reconstitution with adeno-associated viral vectors

12 June 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Effective delivery of large genes to the retina by dual AAV vectors

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Oversized AAV transductifon is mediated via a DNA-PKcs-independent, Rad51C-dependent repair pathway

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Terminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteins

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Lack of correlation between outcomes of membrane repair assay and correction of dystrophic changes in experimental therapeutic strategy in dysferlinopathy

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Native molecular state of adeno-associated viral vectors revealed by single-molecule sequencing

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Ferlins: regulators of vesicle fusion for auditory neurotransmission, receptor trafficking and membrane repair

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Dysferlin overexpression in skeletal muscle produces a progressive myopathy.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Extensive mononuclear infiltration and myogenesis characterize recovery of dysferlin-null skeletal muscle from contraction-induced injuries

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Characterization of genome integrity for oversized recombinant AAV vector

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Effect of genome size on AAV vector packaging

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Impaired recovery of dysferlin-null skeletal muscle after contraction-induced injury in vivo

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

The Mre11/Rad50/Nbs1 complex limits adeno-associated virus transduction and replication

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient whole-body transduction with trans-splicing adeno-associated viral vectors

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Host cell DNA repair pathways in adeno-associated viral genome processing

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Synthetic intron improves transduction efficiency of trans-splicing adeno-associated viral vectors.

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

DNA repair in neurons: so if they don't divide what's to repair?

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Defective membrane repair in dysferlin-deficient muscular dystrophy

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Dysferlin is a plasma membrane protein and is expressed early in human development

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Isolation and characterization of RAD51C, a new human member of the RAD51 family of related genes

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice

24 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Expression of human factor VIII by splicing between dimerized AAV vectors

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermolecular cis activation

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Structural analysis of adeno-associated virus transduction circular intermediates.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Dysferlin associates with the developing T-tubule system in rodent and human skeletal muscle

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445010

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

2 December 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26029720

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Intracellular localization of dysferlin and its association with the dihydropyridine receptor

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26029720

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MTM.2015.9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

PubMed publication ID

26029720

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26029720%20AND%20SRC:MED&resulttype=core&format=json