Last Posted: Feb 13, 2020
- 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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