Abstract: The present application relates to the use of CYP4V2 and RdCVF in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy. The present application also relates to an isolated nucleic acid molecule comprising a polynucleotide encoding CYP4V2 and a polynucleotide encoding RdCVF. The present application also relates to an amino acid sequence encoded by the isolated nucleic acid molecule, a vector comprising the isolated nucleic acid molecule, and a cell comprising the nucleic acid or the vector as well as use thereof in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy.

Abstract: A vector includes a polynucleotide encoding CYP4V2 and a promoter operably linked to the polynucleotide encoding CYP4V2. The vector has a good expression effect, high expression speed, and more stable expression intensity. Cells, pharmaceutical compositions, and kits can include the vector. Methods for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy can include administering the vector to a subject.

Abstract: The present application relates to a fusion protein and use thereof. The fusion protein comprises RdCVF and a signal peptide, wherein the signal peptide is an amino acid sequence set forth in any one of SEQ ID NOs: 27-31 and 33. The present application also relates to an isolated nucleic acid molecule comprising a) a polynucleotide encoding RdCVF and b) a polynucleotide encoding a signal peptide. The present application also provides a vector, a cell, and a pharmaceutical composition containing the nucleic acid molecule, as well as medical use thereof. The fusion protein and/or isolated nucleic acid molecule described herein can effectively increase the secretion of RdCVF.

Abstract: The subject application relates to a method for treating retinitis pigmentosa, comprising the step of: enabling a subject in need thereof to have a functional RHO gene, wherein the functional RHO gene does not comprise a mutation site selected from the group consisting of c.C50T and c.C403T. The subject application also relates to a method for editing the RHO gene, and a composition for treating retinitis pigmentosa in a subject.

Abstract: The present application relates to the use of CYP4V2 and RdCVF in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy. The present application also relates to an isolated nucleic acid molecule comprising a polynucleotide encoding CYP4V2 and a polynucleotide encoding RdCVF. The present application also relates to an amino acid sequence encoded by the isolated nucleic acid molecule, a vector comprising the isolated nucleic acid molecule, and a cell comprising the nucleic acid or the vector as well as use thereof in the manufacture of a medicament for treating, alleviating, and/or preventing a disease or disorder associated with retinal pigment epithelium (RPE) atrophy.