ASO gene therapy for LCA10 due to CEP290 intronic mutation c.2991+1655 A>G

Dr. MacDonald explains how an ASO(antisense oligonucleotide)-based gene therapy works on Leber’s Congenital Amaurosis (LCA) 10 patients with a CEP290 gene deep intronic mutation (c.2991+1655 A>G).

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