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

• Published in: The Journal of clinical investigation Vol. 127; no. 5; pp. 1689 - 1699
• Main Authors: 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: Journal Article
• Language: English
• Published: United States American Society for Clinical Investigation 01.05.2017
• Subjects: ADA deficiency; ADA gene; Adenosine; Adenosine deaminase; Adenosine Deaminase - biosynthesis; Adenosine Deaminase - deficiency; Adenosine Deaminase - genetics; Adolescent; Agammaglobulinemia - enzymology; Agammaglobulinemia - genetics; Agammaglobulinemia - therapy; Age; Analysis; Autografts; Biomedical research; Busulfan; Care and treatment; CD34 antigen; Child; Child, Preschool; Children & youth; Clinical Medicine; Clinical trials; Copy number; Data processing; Enrollments; Enzymes; Female; Gene expression; Gene Expression Regulation, Enzymologic; Gene therapy; Genetic aspects; Genetic engineering; Genetic Therapy; Genetic Vectors; Genetics; Genomes; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Hospitals; Humans; Immune reconstitution; Immunodeficiency; Immunology; Infant; Intravenous administration; Leukocytes (granulocytic); Lymphocytes; Male; Medical screening; Medicine; Mitogens; Pediatrics; Peripheral blood mononuclear cells; Product development; R&D; Research & development; Research centers; Retroviridae; Safety and security measures; Severe combined immunodeficiency; Severe Combined Immunodeficiency - enzymology; Severe Combined Immunodeficiency - genetics; Severe Combined Immunodeficiency - therapy; Stem cell transplantation; Stem cells; Studies; Transduction, Genetic; Transplantation; Transplants & implants; Treatment outcome; Los Angeles California; United States > US; California

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 conditioning (RIC) an...