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Article

Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2009

Cao Ou, Hoffman Brad E, Moghimi Babak, Nayak Sushrusha, Cooper Mario, Zhou Shangzhen, Ertl Hildegund C J, High Katherine A, Herzog Roland W

Abstract excerpt

Immune responses to factor IX (F.IX), a major concern in gene therapy for hemophilia, were analyzed for adeno-associated viral (AAV-2) gene transfer to skeletal muscle and liver as a function of the F9 underlying mutation. Vectors identical to those recently used in clinical trials were administered to four lines of hemophilia B mice on a defined genetic background [C3H/HeJ with deletion of endogenous F9 and...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 19603001
DOI: 10.1038/mt.2009.159
PMCID: PMC2835008

Provenance

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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