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Semin Thromb Hemost 2019; 45(07): 743-750
DOI: 10.1055/s-0039-1688445
DOI: 10.1055/s-0039-1688445
Review Article
Gene Therapy: Paving New Roads in the Treatment of Hemophilia
Funding Dr. Yamaguti-Hayakawa reports personal fees and nonfinancial support from Roche outside the submitted work. Dr. Ozelo reports grants from BioMarin, Pfizer, during the conduct of the study and grants from Novo Nordisk, Shire, Bioverativ, and Roche outside the submitted work.
Weitere Informationen
Publikationsverlauf
Publikationsdatum:
16. Mai 2019 (online)
Abstract
Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questions remain pertaining to its use in specific populations such as those with factor inhibitors, those with underlying liver disease, and pediatric patients. Here, the authors provide an update on viral vector and transgene improvements, review the results of recently published gene therapy clinical trials for hemophilia, and discuss the main challenges facing investigators in the field.
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