Abstract: Provided, in an aspect, is a gene therapy vector, such as for treating retinitis pigmentosa. In an embodiment, a target specific optimization design is performed on a PROM1 gene coding sequence to obtain a nucleotide sequence particularly suitable for efficiently expressing a PROM1 protein in a mammalian cell, and a recombinant AAV virus for expressing a normal human PROM1 protein is constructed. Compared with a coding sequence which is not optimized, the expression level of the optimized PROM1 coding sequence (SEQ ID NO.:1) is increased more than three times. The sequence is particularly suitable for expression in a mammalian cell.

Abstract: Provided is a gene therapy vector for treating a retinitis pigmentosa disease. A targeted specific optimization design is performed on a PROM1 gene coding sequence so as to obtain a nucleotide sequence particularly suitable for efficiently expressing a PROM1 protein in a mammalian (such as human) cell (in particular, a photoreceptor cell), and a recombinant AAV virus for expressing a normal human PROM1 protein is constructed. Compared with a coding sequence which is not optimized, the expression level of the PROM1 coding sequence (SEQ ID NO.:1) performed specific optimization is increased as more than three times. The sequence is particularly suitable for expressing a mammalian (such as human) cell. Eye diseases such as a retinitis pigmentosa disease can be treated efficiently.