(Q40039645)

English

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose

scientific article published on 4 December 2007

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scholarly article

1 reference

Europe PubMed Central

16 September 2017

Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose (English)

1 reference

Europe PubMed Central

16 September 2017

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author name string

Zhijian Wu

1

1 reference

Europe PubMed Central

16 September 2017

Junjiang Sun

2

1 reference

Europe PubMed Central

16 September 2017

Taiping Zhang

3

1 reference

Europe PubMed Central

16 September 2017

Chaoying Yin

4

1 reference

Europe PubMed Central

16 September 2017

Fang Yin

5

1 reference

Europe PubMed Central

16 September 2017

Terry Van Dyke

6

1 reference

Europe PubMed Central

16 September 2017

Richard J Samulski

7

1 reference

Europe PubMed Central

16 September 2017

Paul E Monahan

8

1 reference

Europe PubMed Central

16 September 2017

publication date

4 December 2007

1 reference

Europe PubMed Central

16 September 2017

Molecular Therapy

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Europe PubMed Central

16 September 2017

16

1 reference

Europe PubMed Central

16 September 2017

280-289

1 reference

Europe PubMed Central

16 September 2017

2

1 reference

Europe PubMed Central

16 September 2017

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

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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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

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Adeno-associated virus vectors in clinical trials

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Evaluation of risks related to the use of adeno-associated virus-based vectors.

1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Delivery of MDR1 small interfering RNA by self-complementary recombinant adeno-associated virus vector

1 reference

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7 January 2021

based on heuristic

inferred from DOI database lookup

Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phosphatase vectors as helper viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Efficient gene delivery to human and rodent islets with double-stranded (ds) AAV-based vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Creation of a mouse expressing defective human factor IX

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Globin gene transcripts can utilize histone gene 3' end processing signals

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Efficient expression of protein coding genes from the murine U1 small nuclear RNA promoters

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

A common transcriptional activator is located in the coding region of two replication-dependent mouse histone genes

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Gene therapy expression vectors based on the clotting Factor IX promoter

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Role of intron I in expression of the human factor IX gene

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Inclusion of the NS2-specific exon in minute virus of mice mRNA is facilitated by an intronic splicing enhancer that affects definition of the downstream small intron

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Codon usage limitation in the expression of HIV-1 envelope glycoprotein

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

High guanine and cytosine content increases mRNA levels in mammalian cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

The transcriptional activator hepatocyte nuclear factor 6 regulates liver gene expression

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

The canine factor VIII 3'-untranslated region and a concatemeric hepatocyte nuclear factor 1 regulatory element enhance factor VIII transgene expression in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.MT.6300355

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1038/SJ.MT.6300355

1 reference

Europe PubMed Central

16 September 2017

1 reference

Europe PubMed Central

16 September 2017

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