Last Posted: Aug 16, 2018
- The Next Phase of Human Gene-Therapy Oversight.
Collins Francis S et al. The New England journal of medicine 2018 Aug - Gene therapy for neurological disorders: progress and prospects.
Deverman Benjamin E et al. Nature reviews. Drug discovery 2018 Aug - Gene-silencing technology gets first drug approval after 20-year wait The US Food and Drug Administration's decision breathes new life into RNA-interference therapies.
H Ledford, Nature, August 10, 2018 - The clinical implications of molecular monitoring and analyses of inherited retinal diseases.
Chacón-Camacho Oscar F et al. Expert review of molecular diagnostics 2017 17(11) 1009-1021 - The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.
Flotte Terence R et al. Human gene therapy. Clinical development 2017 Dec 28(4) 178-186 - They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. Experimental gene therapies have yielded promising results in early trials. But the drugs have left some patients wary, worried that success will not last.
G Kolata, New York Times, August 13, 2018 - Gene-silencing technology gets first drug approval after 20-year wait The US Food and Drug Administration's decision breathes new life into RNA-interference therapies.
H Ledford, Nature, August 10, 2018 - Gene therapy for neurological disorders: progress and prospects
BE Deverman et al, Nature Rev Drug Discovery, AUgust 10, 2018 - An Appalachian odyssey: Hunting for ALS genes along a sprawling family tree
E Boodman, Stat News, August 5, 2018 - Management guidelines for paediatric patients receiving chimeric antigen receptor T cell therapy.
Mahadeo Kris M et al. Nature reviews. Clinical oncology 2018 Aug
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