Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

Abstract excerpt

Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as FVIII protein injection and gene therapies via AAV-delivered F8 transgene in an episome are costly and nonpermanent. Here, we describe a CRISPR/Cas9-based in vivo genome editing method, combined with non-homologous end joining, enabling...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to CAR-T cell therapy research
• Share to CRISPR and Genetic Engineering
• Share to Virus-based gene therapy research