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In vitro and In vivo Model Systems for Hemophilia A Gene Therapy

Hemophilia A is a hereditary disorder caused by various mutations in factor VIII gene resulting in either a severe deficit or total lack of the corresponding activity. Recent success in gene therapy of a related disease, hemophilia B, gives new hope that similar success can be achieved for hemophili...

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Detalhes bibliográficos
Publicado no:J Genet Syndr Gene Ther
Main Authors: Mao, Jianhua, Xi, Xiaodong, Kapranov, Philipp, Dong, Biao, Firrman, Jenni, Xu, Ruian, Xiao, Weidong
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4229687/
https://ncbi.nlm.nih.gov/pubmed/25401041
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4172/2157-7412.S1-014
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