COMPOSITIONS AND METHODS FOR MODIFICATION OF SPECIFIED TARGET NUCLEIC ACID SEQUENCE Russian patent published in 2018 - IPC A61K31/7105 C12N15/62 A61K48/00 A61K38/00 

FIELD: biochemistry.

SUBSTANCE: invention relates to biotechnology. Method for modifying a given target site in a target nucleic acid sequence in a host cell using a programmable nucleoprotein molecular complex is described. Programmable polypeptide or nucleic acid sequence encoding a programmable polypeptide is delivered to the host cell. Polypeptide comprises a functional domain capable of modifying said target site devoid of a specific nucleic acid binding site and a binding domain capable of interacting with a specificity contributing nucleic acid (SCNA), where the binding domain is devoid of the specific binding site of the target nucleic acid. Also specificity contributing nucleic acid (SCNA) molecule comprising at least 18 nucleotides, or a nucleic acid encoding SCNA, is delivered. SCNA molecule contains a nucleotide sequence complementary to the target nucleic acid portion and a recognition site capable of specifically attaching to the binding domain of the programmable polypeptide. Presence of a polypeptide in a host cell containing SCNA makes it possible to attach said polypeptide to SCNA, thereby forming an active, programmed nucleoprotein complex. As a result, the active programmed nucleoprotein complex is targeted to the target site in the target nucleic acid sequence in the host cell, which facilitates the modification of the target site by the said active programmed nucleoprotein complex. Composition comprising such a complex and corresponding vectors is also provided.

EFFECT: programmable nucleoprotein molecular complex is capable of specifically modifying and /or correcting the target site in the target nucleic acid sequence and / or modifying the function of the target nucleic acid sequence.

33 cl, 16 dwg, 12 ex