The present disclosure provides an adeno-associated viral (AAV) vector system for expressing a human ABCA4 protein in a target cell, the AAV vector system comprising a first AAV vector comprising a first nucleic acid sequence and a second AAV vector comprising a second nucleic acid sequence； wherein the first nucleic acid sequence comprises a 5′ end portion of an ABCA4 coding sequence (CDS) and the second nucleic acid sequence comprises a 3′ end portion of an ABCA4 CDS, and the 5′ end portion and the 3′ end portion together encompass the entire ABCA4 CDS； wherein the first nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 105 to 3597 of SEQ ID NO: 1； wherein the second nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 3806 to 6926 of SEQ ID NO: 1； wherein the first nucleic acid sequence and the second nucleic acid sequence each comprise a region of sequence overlap with the other； and wherein the region of sequence overlap comprises at least about 20 contiguous nucleotides of a nucleic acid sequence corresponding to nucleotides 3598 to 3805 of SEQ ID NO: 1. Also provided are uses of AAV vector systems in the prevention or treatment of disease.本揭露提供一種在目標細胞中表現人ABCA4蛋白質之腺相關病毒(AAV)載體系統，該AAV載體系統包含第一AAV載體及第二AAV載體，該第一AAV載體包含第一核酸序列且該第二AAV載體包含第二核酸序列；其中該第一核酸序列包含ABCA4編碼序列(CDS)之5'端部分且該第二核酸序列包含ABCA4 CDS之3'端部分，且該5'端部分及該3'端部分一起涵蓋整個ABCA4 CDS；其中該第一核酸序列包含對應SEQ ID NO：1之核苷酸105至3597的毗連核苷酸之序列；其中該第二核酸序列包含對應SEQ ID NO：1之核苷酸3806至6926的毗連核苷酸之序列；其中該第一核酸序列及該第二核酸序列各包含彼此重疊之序列區；且其中該序列重疊區包含對應SEQ ID NO：1之核苷酸3598至3805之核酸序列的至少約20個毗連核苷酸。本揭露亦提供AAV載體系統於預防或治療疾病之用途。
