Gene therapy [NEW TOPIC PAGE]

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Last Posted: Feb 13, 2020

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• Clinical Development on the Frontier: Gene Therapy for Duchenne Muscular Dystrophy.
  Asher Damon R et al. Expert opinion on biological therapy 2020 Feb
• Investigational Hemophilia A Gene Therapy Shows Sustained Benefit
  J Abbasi, JAMA, February 11, 2020
• Lentiviral gene therapy for X-linked chronic granulomatous disease
  DB Kohn et al, Nature Medicine, January 27, 2020
• Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.
  Landrum Peay Holly et al. PloS one 2019 14(5) e0213649
• The impact of exome sequencing on diagnostic yield of muscular dystrophies in consanguineous families.
  Dardas Zain et al. European journal of medical genetics 2020 Jan 103845
• The National Gene Vector Biorepository. Eleven Years of Providing Resources to the Gene Therapy Community.
  Cornetta Kenneth G et al. Human gene therapy 2020 Jan
• At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease
  NY Times, January 11, 2020
• [Advances in newborn screening and immune system reconstitution of severe combined immunodeficiency].
  Huang Shumin et al. Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences 2019 Jun 48(4) 351-357
• The role of hypoxia-inducible factors in neovascular age-related macular degeneration: a gene therapy perspective.
  Mammadzada Parviz et al. Cellular and molecular life sciences : CMLS 2019 Dec
• High throughput newborn screening for aromatic L-amino-acid decarboxylase deficiency by analysis of concentrations of 3-O-methyldopa from dried blood spots.
  Brennenstuhl Heiko et al. Journal of inherited metabolic disease 2019 Dec

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