C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 9/00 (2006.01) C12Q 1/68 (2006.01)
Patent
CA 2348156
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.
L'invention concerne la dégradation de protéines par la voie ubiquitine-protéasome. Plus particulièrement, l'invention concerne la dégradation de protéines dans l'ER, y compris le régulateur de perméabilité transmembranaire de la fibrose kystique (CFTR) par la voie ubiquitine-protéasome. L'invention concerne également des méthodes et compositions d'inhibition de cette dégradation et d'activation de la maturation de .DELTA.F508 dans un CFTR fonctionnel, ainsi que de compréhension du rôle de la perte de fonction du CFTR dans la fibrose kystique. L'invention concerne enfin la découverte de protéines responsables du transfert d'ubiquitine dans .DELTA.F508, CFTR et .alpha.¿1?-AT.
Mbm Intellectual Property Law Llp
Millennium Pharmaceuticals Inc.
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