ADENO-ASSOCIATED VIRUS VIRIONS FOR TRANSFERRING GENES INTO NEURAL CELLS

摘要

PROBLEM TO BE SOLVED: To provide means for transferring target therapeutic genes into neural cells by highly-efficient and simpler means.SOLUTION: A recombinant adeno-associated virus virion comprises a capsomere comprising a protein capable of forming virus virion which is a protein comprising an amino acid sequence in which at least one surface-exposed tyrosine residue is substituted by another amino acid residue, in the amino acid sequence having 90% or more of identity to a specific amino acid sequence; and (b) a polynucleotide packaged into the capsomere which comprises a neural cell specific promoter sequence and a nucleotide sequence operably linked with the promoter sequence. More specifically, a recombinant adeno-associated virus virion for transferring target therapeutic genes into neural cells with high efficiency which is capable of passing through a blood brain barrier, a pharmaceutical composition comprising it, a method for producing it, and a kit, and the like are provided.