| abstract |
A composition for correcting an inversion of the blood coagulation factor VIII (F8) gene, the composition comprising: (a) RNA-guided endonuclease or a nucleotide sequence encoding the RNA-guided endonuclease; and (b) Guide RNA that specifically recognizes the following nucleotide sequences or complementary sequences thereof, or a nucleotide sequence that encodes the guide RNA, the specifically recognized nucleotide sequences being a pair of nucleotide sequences selected from the group consisting in: (i) the nucleotide sequence of SEQ ID NO: 1; (ii) the nucleotide sequence of SEQ ID NO: 2; and (iii) a nucleotide sequence of 15 to 25 bp present in the nucleotide sequence of 562,975 bp between (i) and (ii) in intron 22 of the F8 gene, where (iii) does not have a nucleotide sequence, having a sequence homology of at least 80% with (i) or (ii) in the nucleotide sequence of 562,975 bp between (i) and (ii), and simultaneously, of which the nucleotide sequence 5'- N (G / A) G-3 'is attached downstream. |