The present invention refers to a sgRNA molecule comprising a targeting domain for specifically targeting a SNP in the BEST1
coding region of a
pathologic allele, wherein said targeting domain consists of a sequence selected from the group consisting of SEQ ID NO: 3-8, 41-44, 14-20, 50-52 and 54-55, or a sgRNA molecule combination of specifically defined first and second sgRNA molecules, wherein the first and the second sgRNA molecule each comprise a targeting domain for specifically targeting a SNP in the BEST1
gene coding or non-
coding region of a
pathologic allele. The present invention also refers to a
nucleic acid comprising a sequence that encodes the sgRNA molecule or sgRNA molecule combination and to
nucleic acid combinations. The present invention further relates to a recombinant adenovirus-associated
virus (AAV) comprising the nucleic acids according to the present invention or recombinant AAV combinations. The sgRNA molecule, the
nucleic acid, the recombinant AAV and combinations are useful tools for editing of the target domain in the bestrophin-1 (BEST1)
gene to restore BEST1 channel function by e.g.
CRISPR / Cas9)-based
gene editing. The present invention further relates to the sgRNA molecule, the sgRNA molecule combination, the nucleic acid, the nucleic acid combination, the recombinant AAV and the recombinant AAV combination for use in a method for treatment of the human or
animal body by
surgery or therapy and for use in method of treating or preventing BEST1-related retinopathies, in particular autosomal dominant BEST1-related retinopathies.