Leber congenital amaurosis: Current genetic basis, scope for genetic testing and personalized medicine. Kondkar Altaf A et al. Experimental eye research 2019 189107834

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Last Posted: Feb 27, 2020

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• Leber congenital amaurosis: Current genetic basis, scope for genetic testing and personalized medicine.
  Kondkar Altaf A et al. Experimental eye research 2019 189107834
• The Potential Benefits of Deferred Payment for a Hypothetical Gene Therapy for Congestive Heart Failure: A Cost-Consequence Analysis.
  Hlávka Jakub P et al. Applied health economics and health policy 2020 Feb
• Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
  JC Kapetanovic et al. Nature Medicine, February 24, 2020
• Recent highlights and advances in cardiac gene therapy.
  Oh Jae Gyun et al. Discovery medicine 28(155) 229-235
• 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

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