Optimized mini-dystrophin genes and expression cassettes and their use

摘要

PROBLEM TO BE SOLVED: To provide an AAV vector encoding mini-dystrophin, which can be expressed at a high level in a transduced cell of a subject having DMD while minimizing an immune response to the mini-dystrophin protein. A polynucleotide encoding a mini-dystrophin protein, wherein the polynucleotide is (a) a nucleotide sequence consisting of a specific sequence or at least about 90% identical nucleotide sequence; (b) another A poly comprising a nucleotide sequence consisting of a specific sequence or at least about 90% identical nucleotide sequence; or (c) a nucleotide sequence consisting of yet another specific sequence or at least about 90% identical nucleotide sequence thereof. nucleotide. [Selection diagram] None