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Article

Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Mar 2011

Sabatino Denise E, Lange Amy M, Altynova Ekaterina S, Sarkar Rita, Zhou Shangzhen, Merricks Elizabeth P, Franck Helen G, Nichols Timothy C, Arruda Valder R, Kazazian Haig H

Abstract excerpt

Developing adeno-associated viral (AAV)-mediated gene therapy for hemophilia A (HA) has been challenging due to the large size of the factor VIII (FVIII) complementary DNA and the concern for the development of inhibitory antibodies to FVIII in HA patients. Here, we perform a systematic study in HA dogs by delivering a canine FVIII (cFVIII) transgene either as a single chain or two chains in an AAV vector. An...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 21081906
DOI: 10.1038/mt.2010.240
PMCID: PMC3048175

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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