MODIFICATION OF B-CELLS Russian patent published in 2022 - IPC C12N5/16 C12N5/781 

FIELD: biotechnology; cell biology.

SUBSTANCE: present invention relates to biotechnology and cell biology, in particular to a genetically modified B-cell, a composition for its delivery, a method for the production of protein, and the use of the specified cell in a method for the production of protein. The specified genetically modified B-cell produces inhibitory antibodies against a blood clotting factor or protein, which participates in an autoimmune disease, and it is capable of expressing a transgene (an antibody specific relatively to a B-cell). It contains one or more transgenes and one or more additional modifications, which perform knockout of genes of a B-cell receptor, and/or modify cell interactions in germ centers, and modifications inhibiting suppression of any function of the B-cell, associated with an infection by a pathogen or regulation of malignant tumor. At the same time, the specified transgene is expressed episomal in the specified modified B-cells or is integrated into locus of a safety area, selected from a group consisting of CCR5, Rosa, albumin, AAVS1, TCRA, TCRB.

EFFECT: present invention allows for expansion of the arsenal of means for expression of a desired transgene at a therapeutically significant level in a subject for the treatment of genetic diseases, such as hemophilia, diabetes, diseases with lysosome accumulation, A1AT insufficiency, and malignant tumors.

18 cl, 10 ex, 39 dwg