First CRISPR therapy dosed | Nature Biotechnology
An adult with congenital blindness received the first in vivo CRISPR-based therapy, EDIT-101, targeting a CEP290 gene mutation causing Leber congenital amaurosis. This therapy uses AAV5 with guide RNAs and Cas9 enzyme to correct the mutation, differing from Luxturna, which introduces a correct gene copy for RPE65 mutations.
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First CRISPR therapy dosed | Nature Biotechnology
An adult with congenital blindness received the first in vivo CRISPR-based therapy, EDIT-101, targeting a CEP290 gene mutation causing Leber congenital amaurosis. This therapy uses AAV5 with guide RNAs and Cas9 enzyme to correct the mutation, differing from Luxturna, which introduces a correct gene copy for RPE65 mutations.