Study demonstrates feasibility of therapeutic gene delivery for inherited retinal degeneration in children: vision-research.eu

Researchers have demonstrated the ability to deliver a fully functional copy of the CLN3 gene to stem cells of patients with juvenile NCL, an inherited neurodegenerative disease in which a mutation in the CLN3 gene causes early-onset severe central vision loss.

The gene therapy restored production of CLN3 protein in the stem cell-derived retinal neurons, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website until October 31, 2016.

vision-research.eu - The Gateway to European Vision Research

Study demonstrates feasibility of therapeutic gene delivery for inherited retinal degeneration in children

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