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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Publicat a:Mol Ther Methods Clin Dev
Autors principals: El-Akabawy, Nadia, Rodriguez, Martin, Ramamurthy, Ritu, Rabah, Andrew, Trevisan, Brady, Morsi, Alshaimaa, George, Sunil, Shields, Jordan, Meares, Diane, Farland, Andrew, Atala, Anthony, Doering, Christopher B., Spencer, H. Trent, Porada, Christopher D., Almeida-Porada, Graça
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2020
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7109377/
https://ncbi.nlm.nih.gov/pubmed/32258210
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.001
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