HUMAN PROTEINS INVOLVED IN ENDOPLASMIC RETICULUM PROTEIN DEGRADATION

摘要

The invention relates to degradation of proteins via the ubiquitin-proteasome pathway. More particularly, the invention relates to the degradation of proteins in the ER, including cystic fibrosis transmembrane conductance regulator (CFTR) via the ubiquitin-proteasome pathway. The invention provides methods and compositions for inhibiting such degradation and for promoting the maturation of DELTA F508 into a functional CFTR and for understanding the role of loss of CFTR function in CF. The invention resides in the discovery of human proteins responsible for the ubiquitination of DELTA F508, CFTR, and alpha 1-AT.