Bioengineering hemophilia A–specific microvascular grafts for delivery of full-length factor VIII into the bloodstream

Hemophilia A (HA) is a bleeding disorder caused by mutations in the F8 gene encoding coagulation factor VIII (FVIII). Current treatments are based on regular infusions of FVIII concentrates throughout a patient’s life. Alternatively, viral gene therapies that directly deliver F8 in vivo have shown p...

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Pubblicato in:Blood Adv
Autori principali: Neumeyer, Joseph, Lin, Ruei-Zeng, Wang, Kai, Hong, Xuechong, Hua, Tien, Croteau, Stacy E., Neufeld, Ellis J., Melero-Martin, Juan M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2019
Soggetti:
Gene Therapy
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6929393/
https://ncbi.nlm.nih.gov/pubmed/31851760
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019000848
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