Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

Gene therapy (GT) for adenosine deaminase–deficient severe combined immune deficiency (ADA-SCID) can provide significant long-term benefit when patients are given nonmyeloablative conditioning and ADA enzyme-replacement therapy (ERT) is withheld before autologous transplantation of γ-retroviral vect...

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Detalhes bibliográficos
Main Authors: Carbonaro, Denise A., Jin, Xiangyang, Wang, Xingchao, Yu, Xiao-Jin, Rozengurt, Nora, Kaufman, Michael L., Wang, Xiaoyan, Gjertson, David, Zhou, Yang, Blackburn, Michael R., Kohn, Donald B.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2012
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3488883/
https://ncbi.nlm.nih.gov/pubmed/22833548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-02-408591
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