Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency

BACKGROUND. Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase–deficient (ADA-deficient) SCID when combined with reduced intensity condition...

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Publié dans:J Clin Invest
Auteurs principaux: Shaw, Kit L., Garabedian, Elizabeth, Mishra, Suparna, Barman, Provaboti, Davila, Alejandra, Carbonaro, Denise, Shupien, Sally, Silvin, Christopher, Geiger, Sabine, Nowicki, Barbara, Smogorzewska, E. Monika, Brown, Berkley, Wang, Xiaoyan, de Oliveira, Satiro, Choi, Yeong, Ikeda, Alan, Terrazas, Dayna, Fu, Pei-Yu, Yu, Allen, Fernandez, Beatriz Campo, Cooper, Aaron R., Engel, Barbara, Podsakoff, Greg, Balamurugan, Arumugam, Anderson, Stacie, Muul, Linda, Jagadeesh, G. Jayashree, Kapoor, Neena, Tse, John, Moore, Theodore B., Purdy, Ken, Rishi, Radha, Mohan, Kathey, Skoda-Smith, Suzanne, Buchbinder, David, Abraham, Roshini S., Scharenberg, Andrew, Yang, Otto O., Cornetta, Kenneth, Gjertson, David, Hershfield, Michael, Sokolic, Rob, Candotti, Fabio, Kohn, Donald B.
Format: Artigo
Langue:Inglês
Publié: American Society for Clinical Investigation 2017
Sujets:
Clinical Medicine
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5409097/
https://ncbi.nlm.nih.gov/pubmed/28346229
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI90367
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