Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens

Insufficient expression of factor VIII (fVIII) is a major hurdle in the development of successful nucleic acid treatments for hemophilia. However, we recently showed that under myeloablative and reduced-intensity total body irradiation (TBI) conditioning, transplantation of hematopoietic stem cells...

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Detalhes bibliográficos
Main Authors: Ide, Lucienne M., Gangadharan, Bagirath, Chiang, Kuang-Yueh, Doering, Christopher B., Spencer, H. Trent
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2007
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2018667/
https://ncbi.nlm.nih.gov/pubmed/17569821
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2007-04-082602
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