Last Posted: Apr 29, 2019
- Gene Therapy Leaders Push for Germline Editing Moratorium
Clinical Omics, April 24, 2019 - Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach.
Flotte Terence R et al. Human gene therapy 2018 29(4) 401-402 - A Gene Therapy Breakthrough Could Offer a Treatment for the Rare and Deadly 'Bubble Boy' Disease
J Ducharme, Time, April 18, 2019 - Gene therapy restores immunity in infants with rare immunodeficiency disease
NIH News, April 17, 2019 - Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation- Treatment restores immune-system function in young children with severe disorder.
H Ledford, Nature, April 17, 2019 - Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz Ewelina et al. The New England journal of medicine 2019 Apr 380(16) 1525-1534 - A review of gene- and cell-based therapies for familial hypercholesterolemia.
Hajighasemi Saeideh et al. Pharmacological research 2019 Mar 143119-132 - First gene therapy to treat rare blood disease nears European approval
A Feurstein, Stat News, March 29, 2019 - Hope and challenge: Precision medicine in bladder cancer.
Su Hongwei et al. Cancer medicine 2019 Mar - From clinical observations and molecular dissection to novel therapeutic strategies for primary immunodeficiency disorders.
Ochs Hans D et al. American journal of medical genetics. Part A 2018 Apr 176(4) 784-803
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