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Article

Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A

30 Aug 2018

Abstract excerpt

Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medicine. Following this therapeutic paradigm, we are developing a product candidate, designated CD68-ET3-LV CD34 + , for the treatment of the severe bleeding disorder, hemophilia A. The product consists of autologous CD34 + cells transduced with a human immunodeficiency virus 1–based, monocyte...

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PMID: 30160169
DOI: 10.1089/hum.2018.137

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