G) Show Robust Functional Rescue in Primary Nasal Cultures by Novel CFTR Modulators

Autor: Laselva, Onofrio, McCormack, Jacqueline, Bartlett, Claire, Ip, Wan, Gunawardena, Tarini N. A., Ouyang, Hong, Eckford, Paul D. W., Gonska, Tanja, Moraes, Theo J., Bear, Christine E.

Publikováno v: Journal of Personalized Medicine
Volume 10
Issue 4

The combination therapies ORKAMBITM and TRIKAFTATM are approved for people who have the F508del mutation on at least one allele. In this study we examine the effects of potentiator and corrector combinations on the rare mutation c.3700A>
G. Th

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=multidiscipl::610f9cc92075dc3447014b5d9990bda2

Preclinical Studies of a Rare CF-Causing Mutation in the Second Nucleotide Binding Domain (c.3700A>G) Show Robust Functional Rescue in Primary Nasal Cultures by Novel CFTR Modulators.

Autor: Laselva O; Programme in Molecular Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada.; Department of Physiology, University of Toronto, Toronto, ON M5G 8X4, Canada., McCormack J; Programme in Molecular Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Bartlett C; Programme in Translational Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Ip W; Programme in Translational Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Gunawardena TNA; Programme in Molecular Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Ouyang H; Programme in Translational Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Eckford PDW; Programme in Molecular Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada., Gonska T; Programme in Translational Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada.; Department of Paediatrics, University of Toronto, Toronto, ON M5G 8X4, Canada., Moraes TJ; Programme in Translational Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada.; Department of Paediatrics, University of Toronto, Toronto, ON M5G 8X4, Canada., Bear CE; Programme in Molecular Medicine, Hospital for Sick Children, Toronto, ON M5G 8X4, Canada.; Department of Physiology, University of Toronto, Toronto, ON M5G 8X4, Canada.; Department of Biochemistry, University of Toronto, Toronto, ON M5G 8X4, Canada.

Publikováno v: Journal of personalized medicine [J Pers Med] 2020 Nov 05; Vol. 10 (4). Date of Electronic Publication: 2020 Nov 05.