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US 2026/0226111 A1Published application
Novel capsid protein variant and use thereof
OwnerFei WU, Zhuoyu NI +1inventors
InventorsFei WU, Zhuoyu NI, Fenghua WANG
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Abstract
An capsid protein variants, recombinant AAV viral particles comprising the same, and a method of using the recombinant AAV viral particles for treating an ocular disease, said capsid protein variants comprises the following substitutions as compared to the parental AAV capsid protein VP1: Q464V, A467P, D469N, 1470M, R471A, D472V, S474G, optionally Y500F and/or S501A; said ocular disease is X-linked hereditary retinoschisis.
Claims27
An AAV capsid protein variant comprising an engineered capsid protein VP1 comprising the following amino acid mutation sites relative to a parental AAV capsid protein VP1:
- Q464V, A467P, D469N, 1470M, R471A, D472V, and S474G, wherein the positions are determined with reference to the amino acid sequence position of SEQ ID NO:
- 51;
- optionally, the VP1 further comprises Y500F and/or S501A.
Dependencies are inferred from the claim text.