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

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...

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Publicado en:Hum Gene Ther
Autores principales: Doering, Christopher B., Denning, Gabriela, Shields, Jordan E., Fine, Eli J., Parker, Ernest T., Srivastava, Alok, Lollar, Pete, Spencer, H. Trent
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc., publishers 2018
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Research Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6196756/
https://ncbi.nlm.nih.gov/pubmed/30160169
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2018.137
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