(Q28256353)

1

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Yongping Yue

2

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Yi Lai

3

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Dongsheng Duan

Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes

4

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language of work or name

English

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publication date

January 2008

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published in

Molecular Therapy

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volume

16

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issue

1

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page(s)

124-30

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cites work

1 reference

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7 January 2021

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

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7 January 2021

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

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7 January 2021

CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021

Expression of a truncated cystic fibrosis transmembrane conductance regulator with an AAV5-pseudotyped vector in primates

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7 January 2021

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

1 reference

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

1 reference

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7 January 2021

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

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7 January 2021

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

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021

Hybrid adeno-associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo.

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7 January 2021

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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7 January 2021

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

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7 January 2021

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

1 reference

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7 January 2021

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

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7 January 2021

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

1 reference

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7 January 2021

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain

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7 January 2021

Analysis of splicing parameters in the dystrophin gene: relevance for physiological and pathogenetic splicing mechanisms

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7 January 2021

Gene therapy: the first two decades and the current state-of-the-art

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7 January 2021

Development of multiple cloning site cis-vectors for recombinant adeno-associated virus production

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021

Enhancement of muscle gene delivery with pseudotyped adeno-associated virus type 5 correlates with myoblast differentiation.

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7 January 2021

Consequences of DNA-dependent protein kinase catalytic subunit deficiency on recombinant adeno-associated virus genome circularization and heterodimerization in muscle tissue

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https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021

Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300322

7 January 2021