Last Posted: Feb 09, 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 - Making gene therapy available at the point of care
J Harris, PHG Foundation, October 24, 2016 - Pediatric Sickle Cell Disease - Past Successes and Future Challenges.
Meier Emily Riehm et al. Pediatric research 2016 Oct - Gene therapy: A new chapter.
King Anthony et al. Nature 2016 537(7621) S158-9 - Genetic medicines for CF: Hype versus reality.
Alton Eric W F W et al. Pediatric pulmonology 2016 Oct 51(S44) S5-S17 - Advances in gene therapy for muscular dystrophies.
Abdul-Razak Hayder et al. F1000Research 2016 5 - Gene editing meets gene therapy
Genomic Education Program, UK, September 2016 - Gene therapy for sickle cell disease passes key preclinical test
Harvard Gazettte, September 2016 - Gene editing meets gene therapy
Genomic Education Program, UK, September 2016
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