| abstract |
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 |