Last Posted: Nov 05, 2018
- Thousands of sickle cell and hemophilia patients are waiting for gene therapies. Can manufacturers keep up?
E Mullin, Stat News, November 5, 2018 - Emerging Genetic Therapy for Sickle Cell Disease.
Orkin Stuart H et al. Annual review of medicine 2018 Oct - Genetic screening of Russian Usher syndrome patients toward selection for gene therapy.
Ivanova Marianna E et al. Ophthalmic genetics 2018 Oct 1-8 - RNAi Rollercoaster Begins to Reap Therapies
BM Kuehn, JAMA, October 30, 2018 - Two sick children and a $1.5 million bill: One family’s race for a gene therapy cure
A Regalado, Tech Review, October 23, 2018 - Promise of Precision Medicine in Psychiatry Nears Reality
L Davenport, Medscape, October 1o, 2018 - Results of 5-year follow-up study in patients with peripheral artery disease treated with PL-VEGF165 for intermittent claudication.
Deev Roman et al. Therapeutic advances in cardiovascular disease 2018 Sep 12(9) 237-246 - The Next Phase of Human Gene-Therapy Oversight
F Collins et al, NEJM, October 10, 2018 - T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.
Clarke Erik L et al. Genome medicine 2018 Sep (1) 70 - Challenges and Advances in Gene Therapy Approaches for Neurodegenerative Disorders.
Donde Aneesh et al. Current gene therapy 2017 17(3) 187-193
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