COMPOSITIONS AND METHODS FOR DEGRADATION OF IMPROPERLY PACKAGED PROTEINS Russian patent published in 2021 - IPC A61K38/21 A61P11/00 A61P25/28 G01N33/566 

FIELD: medicine.

SUBSTANCE: group of inventions relates to methods for enhancing degradation of improperly packaged proteins and protein aggregates. A composition for the treatment or prevention of a disease or disorder associated with an improperly packaged protein or protein aggregates contains a vector based on an adeno associated virus (AAV) containing a nucleic acid sequence encoding human TRIM11, where the specified disease or disorder is neurodegenerative disease or disorder. A method for the treatment or prevention of a disease or disorder associated with an improperly packaged protein or protein aggregates in a subject who needs it, a composition, and a method for the treatment or prevention of a disease or disorder associated with degradation of a functional mutant protein, where the specified disease or disorder is cystic fibrosis, and a method for obtaining a recombinant protein are also proposed.

EFFECT: enhancing degradation of improperly packaged proteins and protein aggregates.

9 cl, 42 dwg, 4 tbl, 5 ex