Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy (Q44405190)

2 December 2017

title

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy (English)

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2 December 2017

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Vandendriessche T

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Thorrez L

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Acosta-Sanchez A

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Petrus I

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Wang L

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Ma L

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DE Waele L

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Iwasaki Y

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Gillijns V

9

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Wilson JM

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Collen D

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Chuah MK

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publication date

26 September 2006

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Journal of Thrombosis and Haemostasis

2 December 2017

published in

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volume

5

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issue

1

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page(s)

16-24

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Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

2 December 2017

cites work

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https://api.crossref.org/works/10.1111/J.1538-7836.2006.02220.X

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

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

21 January 2018

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https://api.crossref.org/works/10.1111/J.1538-7836.2006.02220.X

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

21 January 2018

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https://api.crossref.org/works/10.1111/J.1538-7836.2006.02220.X

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

21 January 2018

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A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

21 January 2018

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Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

21 January 2018

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

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

21 January 2018

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

21 January 2018

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Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

21 January 2018

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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

21 January 2018

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Efficient selection for high-expression transfectants with a novel eukaryotic vector

21 January 2018

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Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

21 January 2018

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A factor IX-deficient mouse model for hemophilia B gene therapy

21 January 2018

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Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males

21 January 2018

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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

21 January 2018

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

21 January 2018

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

21 January 2018

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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

21 January 2018

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Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

21 January 2018

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Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

21 January 2018

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Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

21 January 2018

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Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

21 January 2018

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Targeting ischemic cardiac dysfunction through gene transfer

21 January 2018

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Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

21 January 2018

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

21 January 2018

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

21 January 2018

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21 January 2018

Identifiers

DOI

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2 December 2017

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