The present disclosure provides methods for treating Cystic Fibrosis in a subject by administering to the subject a corrector agent capable of acting through MSD1 during the biosynthesis of CFTR protein. The disclosure also provides methods of screening for new corrector agents capable of acting through MSD1 during the biosynthesis of a CFTR protein.
申请公布号
WO2014144860(A1)
申请公布日期
2014.09.18
申请号
WO2014US29449
申请日期
2014.03.14
申请人
VERTEX PHARMACEUTICALS INCORPORATED;THE UNIVERSITY OF NORTH CAROLINA CHAPEL HILL
发明人
VAN GOOR, FREDRICK, F.;HOFFMAN, BETH, JENNIFER;DELA ROSA, OXANA, ADOLFOVNA;CYR, DOUGLAS