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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Vydáno v:J Clin Invest
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society for Clinical Investigation 2017
Témata:
Clinical Medicine
On-line přístup: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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