Last Posted: Dec 10, 2017
- A Milestone for CAR T Cells
E Tran et al, NEJM, Dec 10, 2017 - Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George Lindsey A et al. The New England journal of medicine 2017 Dec (23) 2215-2227 - Ethical considerations in gene therapy.
Levin Alex V et al. Ophthalmic genetics 2016 37(3) 249-51 - A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.
Cai Liuhong et al. Stem cells translational medicine 2017 Nov - Contemporary genetic testing in inherited cardiac disease: tools, ethical issues, and clinical applications.
Girolami Francesca et al. Journal of cardiovascular medicine (Hagerstown, Md.) 2017 Nov - First-In-Human Phase 1 CRISPR Gene Editing Cancer Trials: Are We Ready?
Baylis Francoise et al. Current gene therapy 2017 Nov - Gene Therapy Hits a Peculiar Roadblock: A Virus Shortage
G Kolata, NY Times, Nov 27, 2017 - Gene Therapy Shows Promise For A Growing List Of Diseases
R Stein, NPR, Nov 29, 2017 - The Promise and Challenges of CAR-T Gene Therapy.
Kuehn Bridget M et al. JAMA 2017 Nov - The Promise and Challenges of CAR-T Gene Therapy
BM Kuehn, JAMA, Nov 22, 2017 - Gene therapy: evidence, value and affordability in the US health care system.
Hampson Grace et al. Journal of comparative effectiveness research 2017 Nov - Roadmap for an early gene therapy for cystic fibrosis airway disease.
Carlon Marianne S et al. Prenatal diagnosis 2017 Oct - Gene therapy creates boy’s replacement skin from his stem cells
R Lewis, Genetic Literacy Project, Nov 8, 2017 - Gene Therapy Creates Replacement Skin to Save a Dying Boy
D Grady, NY Times, Nov 8, 2017 - Gene therapy: Transgenic stem cells replace skin
M Aragona et al, Nature News, November 7, 2017 - The Dilemma of Two Innovative Therapies for Spinal Muscular Atrophy.
van der Ploeg Ans T et al. The New England journal of medicine 2017 11 (18) 1786-1787
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