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Article

Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene.

Gene therapy - 1 Nov 2017

Staber J M, Pollpeter M J, Anderson C-G, Burrascano M, Cooney A L, Sinn P L, Rutkowski D T, Raschke W C, McCray P B

Abstract excerpt

Current therapies for hemophilia A include frequent prophylactic or on-demand intravenous factor treatments which are costly, inconvenient and may lead to inhibitor formation. Viral vector delivery of factor VIII (FVIII) cDNA has the potential to alleviate the debilitating clotting defects. Lentiviral-based vectors delivered to murine models of hemophilia A mediate phenotypic correction. However, a limitation of...

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Identifiers

PMID: 28905885
DOI: 10.1038/gt.2017.67
PMCID: PMC5937993

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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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