Last Posted: Mar 23, 2017
- Gene Therapy May Finally Be Coming of Age
KQED Science, March 20, 2017 - Gene delivery to the lungs: pulmonary gene therapy for cystic fibrosis.
Villate-Beitia Ilia et al. Drug development and industrial pharmacy 2017 Mar 1-11 - Recent advances in hemophilia B therapy.
Horava Sarena D et al. Drug delivery and translational research 2017 Feb - Rounding up sickle cells with gene therapy
L Byrne, Science Translational Medicine, March 15, 2017 - Gene Therapy in a Patient with Sickle Cell Disease
Ramya Ramaswami, Resident 360 NEJM Blog, March 2017 - 10 Years of Rare Disease Day.
Yáñez-Muñoz R J et al. Gene therapy 2017 Feb 24(2) 67 - Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil Jean-Antoine et al. The New England journal of medicine 2017 Mar 376(9) 848-855 - 10 Years of Rare Disease Day
RJY Munoz, Gene Therapy, February 2017 - Gene therapy: Deaf to hearing a whisper
J Gallagher, BBC, February 7, 2017 - FDA Gives Breakthrough Designation to Hemophilia Gene Therapy
National Hemophilia Foundation, January 30, 2017 - Everything You Need to Know About Gene Therapy’s Most Promising Year
A Regalado, MIT Technology Review, December 30, 2016 - Snapshots of Life: Lighting up the Promise of Retinal Gene Therapy
Francis Collins, NIH Director, November 10, 2016 - Gene therapy for blistering skin disease appears to enhance healing in clinical trial
Stanford Medicine, November 1, 2016 - Overview of 15-year severe combined immunodeficiency in the Netherlands: towards newborn blood spot screening.
de Pagter Anne P J et al. European journal of pediatrics 2015 Sep 174(9) 1183-8 - Regulatory Oversight of Cell and Gene Therapy Products in Canada.
Ridgway Anthony et al. Advances in experimental medicine and biology 2015 87149-71 - Gene therapy shows promise for treating Niemann-Pick disease type C1
NIH, October 26, 2016
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