GENE THERAPY

Public Health Genomics Knowledge Base (v1.2)


Last Posted: Feb 09, 2017

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• 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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