Article

Improved Intrvenous Lentiviral Gene Therapy Based on Endothelial-specific Factor VIII for Hemophilia A

2023-03-13

Abstract excerpt

<title>Abstract</title> <p><bold>Background:</bold> Hemophilia A (HA) is an X-linked monogenic disorder caused by deficiency of the factor VIII (FVIII, F8) gene in the intrinsic coagulation cascade. The current protein replacement therapy (PRT) of HA has many limitations including short term effectiveness, high cost, and life-time treatment requirement. Gene therapy has become a promising treatment for HA. Orthot...

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Literature Corpus work: d55332de-413a-54de-87f7-f73da5a17dfb
DOI: 10.21203/rs.3.rs-2637226/v1

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