Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants.
Scientific Reports
Phuan, Puay-Wah PW; Tan, Joseph-Anthony JA; Rivera, Amber A AA; Zlock, Lorna L; Nielson, Dennis W DW; Finkbeiner, Walter E WE; Haggie, Peter M PM; Verkman, Alan S AS
Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences.
Human Molecular Genetics
Xue, Xiaojiao X; Mutyam, Venkateshwar V; Thakerar, Amita A; Mobley, James J; Bridges, Robert J RJ; Rowe, Steven M SM; Keeling, Kim M KM; Bedwell, David M DM