Gene therapy [NEW TOPIC PAGE]

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Last Posted: Aug 16, 2019

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• Innovative Therapies for Cystic Fibrosis: The Road from Treatment to Cure.
  Cabrini Giulio et al. Molecular diagnosis & therapy 2019 23(2) 263-279
• Recent progress in translational cystic fibrosis research using precision medicine strategies.
  Cholon Deborah M et al. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society 2018 17(2S) S52-S60
• This Drug Will Save Children’s Lives. It Costs $2 Million.
  NY Times editorial board, August 13, 2019
• Progress and challenges of gene therapy for Pompe disease.
  Ronzitti Giuseppe et al. Annals of translational medicine 2019 Jul 7(13) 287
• The German National Registry of Primary Immunodeficiencies (2012-2017).
  El-Helou Sabine M et al. Frontiers in immunology 2019 101272
• Gene therapy for primary immunodeficiency.
  Booth Claire et al. Human molecular genetics 2019 Jul
• Gene Therapy.
  High Katherine A et al. The New England journal of medicine 2019 Aug (5) 455-464
• Advances In Gene Therapy For Cystic Fibrosis Lung Disease.
  Yan Ziying et al. Human molecular genetics 2019 Jul
• The national blueprint for future basic and translational research to understand factor VIII immunogenicity: NHLBI State of the Science Workshop on factor VIII inhibitors.
  Meeks Shannon L et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Jul 25(4) 595-602
• How do you toast a gene therapy trial? With tea.
  T Carey, NHGRI, July 23, 2019

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