Patent ID: 11965174
Assignee: CITY OF HOPE
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 7:
8. A replication-defective AAV comprising:
(a) a correction genome comprising (i) an editing element for integration into a target locus of a chromosome in a cell, the editing element comprising a coding sequence of a gene optionally operably linked to an exogenous promoter, (ii) a 5′ homologous arm nucleotide sequence 5′ of the editing element, having homology to a 5′ region of the chromosome relative to the target locus, and (iii) a 3′ homologous arm nucleotide sequence 3′ of the editing element, having homology to a 3′ region of the chromosome relative to the target locus; and
(b) an AAV capsid comprising an AAV capsid protein comprising the amino acid sequence of amino acids 138-736 of SEQ ID NO: 13.