METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS AND DISEASES RELATED TO RDH12 Russian patent published in 2022 - IPC C12N15/63 C12N15/86 A61K48/00 

FIELD: biotechnology; medicine.

SUBSTANCE: invention relates to biotechnology and medicine, namely to the correction of non-functional, defective or inadequately expressed native RDH12. Codon-optimized nucleotide sequences for RDH12 are proposed, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing a codon-optimized sequence for functional RDH12. Recombinant vectors that express codon-optimized functional RDH12 are proposed. Compositions containing these codon-optimized sequences are proposed, suitable for use in methods for treating, slowing down or stopping specific blindness-causing diseases resulting due to the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are proposed for the correction of non-functional, defective or inadequately expressed native RDH12.

EFFECT: invention makes it possible to slow down or stop specific blindness-causing diseases that occur due to the absence, deficiency or inappropriate expression of RDH12.

14 cl, 10 dwg, 1 tbl, 5 ex