Recombinant adeno-associated vectors for targeted treatment
AssigneeCity of Hope
InventorsSaswati Chatterjee, Laura Jane Smith, Kamehameha Wong
Life of this patent
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Abstract
Novel adeno-associated virus (AAV) vectors in nucleotide and amino acid forms and uses thereof are provided. The isolates show specific tropism for certain target tissues, such as blood stem cells, liver, heart and joint tissue, and may be used to transduce stem cells for introduction of genes of interest into the target tissues. Certain of the vectors are able to cross tightly controlled biological junctions, such as the blood-brain barrier, which open up additional novel uses and target organs for the vectors, providing for additional methods of gene therapy and drug delivery.
Claims33
A recombinant adeno-associated virus (rAAV) vector comprising a capsid protein, the capsid protein comprising an amino acid sequence having at least 95% sequence identity with the amino acid sequence of amino acids 203-736 of SEQ ID NO:12, wherein:
- the amino acid in the capsid protein corresponding to amino acid 296 of SEQ ID NO:12 is H;
- the amino acid in the capsid protein corresponding to amino acid 464 of SEQ ID NO:12 is N;
- the amino acid in the capsid protein corresponding to amino acid 501 of SEQ ID NO:12 is I;
- the amino acid in the capsid protein corresponding to amino acid 505 of SEQ ID NO:12 is R;
- the amino acid in the capsid protein corresponding to amino acid 681 of SEQ ID NO:12 is M;
- the amino acid in the capsid protein corresponding to amino acid 687 of SEQ ID NO:12 is R;
- or the amino acid in the capsid protein corresponding to amino acid 706 of SEQ ID NO:12 is C.
A rAAV vector comprising a capsid protein, the capsid protein comprising an amino acid sequence having at least 95% sequence identity with the amino acid sequence of amino acids 138-736 of SEQ ID NO:12, wherein:
- the amino acid in the capsid protein corresponding to amino acid 296 of SEQ ID NO:12 is H;
- the amino acid in the capsid protein corresponding to amino acid 464 of SEQ ID NO:12 is N;
- the amino acid in the capsid protein corresponding to amino acid 501 of SEQ ID NO:12 is I;
- the amino acid in the capsid protein corresponding to amino acid 505 of SEQ ID NO:12 is R;
- the amino acid in the capsid protein corresponding to amino acid 681 of SEQ ID NO:12 is M;
- the amino acid in the capsid protein corresponding to amino acid 687 of SEQ ID NO:12 is R;
- or the amino acid in the capsid protein corresponding to amino acid 706 of SEQ ID NO:12 is C.
A rAAV vector comprising a capsid protein, the capsid protein comprising an amino acid sequence having at least 95% sequence identity with the amino acid sequence of SEQ ID NO:12, wherein:
- the amino acid in the capsid protein corresponding to amino acid 296 of SEQ ID NO:12 is H;
- the amino acid in the capsid protein corresponding to amino acid 464 of SEQ ID NO:12 is N;
- the amino acid in the capsid protein corresponding to amino acid 501 of SEQ ID NO:12 is I;
- the amino acid in the capsid protein corresponding to amino acid 505 of SEQ ID NO:12 is R;
- the amino acid in the capsid protein corresponding to amino acid 681 of SEQ ID NO:12 is M;
- the amino acid in the capsid protein corresponding to amino acid 687 of SEQ ID NO:12 is R;
- or the amino acid in the capsid protein corresponding to amino acid 706 of SEQ ID NO:12 is C.
Dependencies are inferred from the claim text.