FIELD: biotechnology.
SUBSTANCE: polynucleotide containing a nucleotide sequence encoding polypeptide PPT1 or its fragment, an expression cassette, an expression vector, a transformed cell, a pharmaceutical composition, methods for their application for the delivery of ORF CLN1 to a cell or a subject, and a method for the treatment of a disorder associated with aberrant expression of CLN1 gene or aberrant activity of CLN1 gene product in a subject are presented.
EFFECT: invention makes it possible to obtain polynucleotide that contains an optimized open frame of CLN1 reading and that is used for the treatment of infantile neuronal lipofuscinosis (infantile Batten disease).
39 cl, 14 dwg, 3 tbl, 1 ex
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