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Article

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

PloS one - 1 Jan 2016

Callan Mary Beth, Haskins Mark E, Wang Ping, Zhou Shangzhen, High Katherine A, Arruda Valder R

Abstract excerpt

Severe hemophilia A (HA) is an inherited bleeding disorder characterized by <1% of residual factor VIII (FVIII) clotting activity. The disease affects several mammals including dogs, and, like humans, is associated with high morbidity and mortality. In gene therapy using adeno-associated viral (AAV) vectors, the canine model has been one of the best predictors of the therapeutic dose tested in clinical trials for...

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Identifiers

PMID: 27011017
DOI: 10.1371/journal.pone.0151800
PMCID: PMC4807047

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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