(Q36384464)

13 August 2017

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title

Gene therapy progress and prospects--vectorology: design and production of expression cassettes in AAV vectors (English)

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13 August 2017

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Le Bec C

1

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13 August 2017

Douar AM

series ordinal

2

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13 August 2017

publication date

1 May 2006

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13 August 2017

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13 August 2017

volume

13

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13 August 2017

issue

10

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13 August 2017

page(s)

805-813

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3302724

13 August 2017

exact match

0 references

cites work

Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

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Clades of Adeno-associated viruses are widely disseminated in human tissues.

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AAV hybrid serotypes: improved vectors for gene delivery

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From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy

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A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia.

21 January 2018

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Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer

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Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo

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Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector

21 January 2018

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AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype

21 January 2018

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Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy

21 January 2018

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The recombinant adeno-associated virus vector (rAAV2)-mediated apolipoprotein B mRNA-specific hammerhead ribozyme: a self-complementary AAV2 vector improves the gene expression

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Gene therapy progress and prospects: transcription regulatory systems.

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Regulatable gene expression systems for gene therapy applications: progress and future challenges

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Tight regulation from a single tet-off rAAV vector as demonstrated by flow cytometry and quantitative, real-time PCR.

21 January 2018

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Optimal design of a single recombinant adeno-associated virus derived from serotypes 1 and 2 to achieve more tightly regulated transgene expression from nonhuman primate muscle

21 January 2018

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Tetracycline-inducible transgene expression mediated by a single AAV vector.

21 January 2018

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Dimerizer regulation of AADC expression and behavioral response in AAV-transduced 6-OHDA lesioned rats

21 January 2018

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Long-term inducible gene expression in the eye via adeno-associated virus gene transfer in nonhuman primates.

21 January 2018

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Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

21 January 2018

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Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

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Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy

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Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer

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Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

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Exploiting internal ribosome entry sites in gene therapy vector design.

21 January 2018

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Light-activated gene transduction of recombinant adeno-associated virus in human mesenchymal stem cells

21 January 2018

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Stable antibody expression at therapeutic levels using the 2A peptide

21 January 2018

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Therapeutic antibody gene transfer.

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Comparison of gene expression after intraperitoneal delivery of AAV2 or AAV5 in utero

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

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

21 January 2018

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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

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21 January 2018

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Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adeno-associated virus genomes

21 January 2018

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Exogenous control of mammalian gene expression through modulation of RNA self-cleavage

21 January 2018

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Use of adeno-associated viral vector for delivery of small interfering RNA.

21 January 2018

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Inhibition of NF-kappaB mediated inflammation by siRNA expressed by recombinant adeno-associated virus

21 January 2018

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Attenuation of dengue virus infection by adeno-associated virus-mediated siRNA delivery

21 January 2018

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In vivo inhibition of hippocampal Ca2+/calmodulin-dependent protein kinase II by RNA interference.

21 January 2018

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RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

21 January 2018

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21 January 2018

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21 January 2018

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

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Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

21 January 2018

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Genomic stability of self-complementary adeno-associated virus 2 during early stages of transduction in mouse muscle in vivo

21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Analysis of site-specific transgene integration following cotransduction with recombinant adeno-associated virus and a rep encodingplasmid

21 January 2018

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Adeno-associated virus: from defective virus to effective vector

21 January 2018

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Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors.

21 January 2018

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Gene targeting with viral vectors

21 January 2018

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Facile methods for generating human somatic cell gene knockouts using recombinant adeno-associated viruses

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

Human Gene Targeting by Adeno-Associated Virus Vectors Is Enhanced by DNA Double-Strand Breaks

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

Gene targeting in stem cells from individuals with osteogenesis imperfecta

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

Strain-specific rate of shutdown of CMV enhancer activity in murine liver confirmed by use of persistent [E1(-), E2b(-)] adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

inferred from DOI database lookup

7 January 2021

based on heuristic

Adeno-associated vector mediated gene transfer of transforming growth factor-beta1 to normal and osteoarthritic human chondrocytes stimulates cartilage anabolism

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

inferred from DOI database lookup

7 January 2021

based on heuristic

Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302724

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/SJ.GT.3302724

1 reference

13 August 2017

1 reference