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
The combination therapies ORKAMBI<sup>TM</sup> and TRIKAFTA<sup>TM</sup> 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. This mutati...
Main Authors: | Onofrio Laselva, Jacqueline McCormack, Claire Bartlett, Wan Ip, Tarini N. A. Gunawardena, Hong Ouyang, Paul D. W. Eckford, Tanja Gonska, Theo J. Moraes, Christine E. Bear |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2020-11-01
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Series: | Journal of Personalized Medicine |
Subjects: | |
Online Access: | https://www.mdpi.com/2075-4426/10/4/209 |
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