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Therapeutic Gene Delivery for Inherited Retinal Degeneration in Children

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.
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