Last Posted: Feb 27, 2020
- 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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