Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose (Q40039645)

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scientific article published on 4 December 2007

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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

PubMed publication ID

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

PubMed publication ID

16 September 2017

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author name string

Zhijian Wu

1

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Europe PubMed Central

PubMed publication ID

16 September 2017

Junjiang Sun

2

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Europe PubMed Central

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16 September 2017

Taiping Zhang

3

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

Chaoying Yin

4

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

Fang Yin

5

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

Terry Van Dyke

6

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Europe PubMed Central

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16 September 2017

Richard J Samulski

7

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

Paul E Monahan

8

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Europe PubMed Central

PubMed publication ID

16 September 2017

publication date

4 December 2007

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Europe PubMed Central

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16 September 2017

Molecular Therapy

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Europe PubMed Central

PubMed publication ID

16 September 2017

16

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Europe PubMed Central

PubMed publication ID

16 September 2017

2

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Europe PubMed Central

PubMed publication ID

16 September 2017

280-289

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Europe PubMed Central

PubMed publication ID

16 September 2017

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

1 reference

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

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7 January 2021

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

1 reference

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7 January 2021

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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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7 January 2021

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Adeno-associated virus vectors in clinical trials

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7 January 2021

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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

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

1 reference

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based on heuristic

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

1 reference

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

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

1 reference

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based on heuristic

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

1 reference

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7 January 2021

based on heuristic

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

1 reference

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7 January 2021

based on heuristic

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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

1 reference

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

1 reference

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

1 reference

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

1 reference

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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

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

1 reference

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7 January 2021

based on heuristic

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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

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Creation of a mouse expressing defective human factor IX

1 reference

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7 January 2021

based on heuristic

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

1 reference

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7 January 2021

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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

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7 January 2021

based on heuristic

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

1 reference

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7 January 2021

based on heuristic

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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

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7 January 2021

based on heuristic

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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

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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

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7 January 2021

based on heuristic

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

1 reference

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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

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7 January 2021

based on heuristic

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

1 reference

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7 January 2021

based on heuristic

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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

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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

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7 January 2021

based on heuristic

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Posttranslational modifications of recombinant myotube-synthesized human factor IX.

1 reference

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7 January 2021

based on heuristic

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

1 reference

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7 January 2021

based on heuristic

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The transcriptional activator hepatocyte nuclear factor 6 regulates liver gene expression

1 reference

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7 January 2021

based on heuristic

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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

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7 January 2021

based on heuristic

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Identifiers

10.1038/SJ.MT.6300355

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

16 September 2017

ResearchGate publication ID

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