Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A

The delivery of factor VIII (FVIII) through gene and/or cellular platforms has emerged as a promising hemophilia A treatment. Herein, we investigated the suitability of human placental cells (PLCs) as delivery vehicles for FVIII and determined an optimal FVIII transgene to produce/secrete therapeuti...

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Bibliographic Details
Main Authors: Nadia El-Akabawy, Martin Rodriguez, Ritu Ramamurthy, Andrew Rabah, Brady Trevisan, Alshaimaa Morsi, Sunil George, Jordan Shields, Diane Meares, Andrew Farland, Anthony Atala, Christopher B. Doering, H. Trent Spencer, Christopher D. Porada, Graça Almeida-Porada
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hemophilia A
FVIII
gene therapy
cell therapy
ET3
HSQ
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300310

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