Gene Therapy: Paving New Roads in the Treatment of Hemophilia

Gabriela G. Yamaguti-Hayakawa; Margareth C. Ozelo

doi:10.1055/s-0039-1688445

Seminars in Thrombosis and Hemostasis, Inhaltsverzeichnis

Semin Thromb Hemost 2019; 45(07): 743-750
DOI: 10.1055/s-0039-1688445

Review Article

Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Gene Therapy: Paving New Roads in the Treatment of Hemophilia

Gabriela G. Yamaguti-Hayakawa

¹Department of Internal Medicine, Faculty of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil

²IHTC Hemophilia Unit Cláudio Luiz Pizzigatti Corrêa, INCT do Sangue Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil

,

Margareth C. Ozelo

¹Department of Internal Medicine, Faculty of Medical Sciences, University of Campinas, Campinas, São Paulo, Brazil

²IHTC Hemophilia Unit Cláudio Luiz Pizzigatti Corrêa, INCT do Sangue Hemocentro UNICAMP, University of Campinas, Campinas, São Paulo, Brazil

› Institutsangaben

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.

Keywords

hemophilia - factor VIII - factor IX - gene therapy - AAV vector

Volltext

Referenzen

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