T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34(+) cells in ADA-deficient SCID neonates

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for gene-corrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this diseas...

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Main Authors: Kohn, Donald B., Hershfield, Michael S., Carbonaro, Denise, Shigeoka, Ann, Brooks, Judith, Smogorzewska, E. Monika, Barsky, Lora W., Chan, Raymond, Burotto, Felix, Annett, Geralyn, Nolta, Jan A., Crooks, Gay, Kapoor, Neena, Elder, Melissa, Wara, Diane, Bowen, Thomas, Madsen, Edward, Snyder, Floyd F., Bastian, John, Muul, Linda, Blaese, R. Michael, Weinberg, Kenneth, Parkman, Robertson
Format: Artigo
Language:Inglês
Published: 1998
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3777239/
https://ncbi.nlm.nih.gov/pubmed/9662367
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