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Human Gene Therapy|March 23, 2013
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012Marina O'Reilly, Donald B Kohn, Jeffrey Bartlett, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 15, 2022
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trialsKenneth Cornetta, Jing Yao, Kimberley House, et al.
The New England Journal of Medicine|October 15, 2025
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase DeficiencyClaire Booth, Katelyn Masiuk, Konstantinos Vazouras, et al.
Nature|September 17, 2010
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaMarina Cavazzana-Calvo, Emmanuel Payen, Olivier Negre, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|March 18, 2014
Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanomaThinle Chodon, Begoña Comin-Anduix, Bartosz Chmielowski, et al.
Blood|May 11, 2021
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiencyBryanna Reinhardt, Omar Habib, Kit L Shaw, et al.
The Journal of Clinical Investigation|March 28, 2017
Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiencyKit L Shaw, Elizabeth Garabedian, Suparna Mishra, et al.
The New England Journal of Medicine|May 11, 2021
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase DeficiencyDonald B Kohn, Claire Booth, Kit L Shaw, et al.
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