FIELD: biotechnology.
SUBSTANCE: described is a vector system based on an adeno-associated virus (AAV) for expression of a protein of an ATP-binding cassette, a subfamily A, member 4 (ABCA4) of a human in a target cell, containing a first AAV vector containing a first nucleic acid sequence and a second AAV vector containing a second nucleic acid sequence; wherein the first nucleic acid sequence comprises a 5'-terminal part of the ABCA4 coding sequence (CDS) and the second nucleic acid sequence comprises a 3'-terminal part of the ABCA4 CDS, and the 5'-terminal part and the 3'-terminal part together include the whole ABCA4 CDS; wherein the first nucleic acid sequence comprises a sequence of adjacent nucleotides corresponding to nucleotides 105-3597 SEQ ID NO: 1 or SEQ ID NO: 2; wherein the second nucleic acid sequence comprises a sequence of adjacent nucleotides corresponding to nucleotides 3806-6926 SEQ ID NO: 1 or SEQ ID NO: 2; wherein each of the first nucleic acid sequence and the second nucleic acid sequence comprises an overlap area of the sequences; and wherein the overlap area of the sequences comprises at least approximately 20 adjacent nucleotides from the nucleic acid sequence corresponding to nucleotides 3598-3805 SEQ ID NO: 1 or SEQ ID NO: 2. The present invention also relates to an application of AAV vector systems in prevention or treatment of a disease characterised by degradation of retinal cells, preferably, Stargardt disease.
EFFECT: invention expands the range of means for treating diseases characterised by degradation of retinal cells.
22 cl, 12 dwg, 6 ex
