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Hemophilia Gene Therapy: From Bench to Bed Side.

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Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete… Click to show full abstract

Novel gene therapy strategies have changed the prognosis of many inherited diseases in recent years. New development in genetic tools and study models has brought us closer to a complete cure for hemophilia. This review will address the latest gene therapy research in hemophilia A and B including gene therapy tools, genetic strategies and animal models, and summarizes results of recent clinical trials. We will also discuss potential solutions to the current barriers in gene therapy for hemophilia.

Keywords: gene therapy; therapy bench; hemophilia gene; bench bed; gene

Journal Title: Current gene therapy
Year Published: 2019

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