Submitted by Anonymous (not verified) on 5 June 2025 - 14:10
Orphan designation: recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene Treatment of Leber's congenital amaurosis, 16/10/2017 Withdrawn
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