F
Fredrick Van Goor
Researcher at Vertex Pharmaceuticals
Publications - 65
Citations - 6920
Fredrick Van Goor is an academic researcher from Vertex Pharmaceuticals. The author has contributed to research in topics: Cystic fibrosis transmembrane conductance regulator & Cystic fibrosis. The author has an hindex of 30, co-authored 64 publications receiving 6151 citations. Previous affiliations of Fredrick Van Goor include National Institutes of Health & University of Alberta.
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Journal ArticleDOI
Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770
Fredrick Van Goor,Sabine Hadida,Peter D. J. Grootenhuis,B. Burton,Dong Cao,Tim Neuberger,Amanda Turnbull,Ashvani K. Singh,John Joubran,Anna R. Hazlewood,Jinglan Zhou,Mccartney Jason,Vijayalaksmi Arumugam,Caroline Decker,Jennifer Yang,Christopher Young,Eric R. Olson,Jeffery J. Wine,Raymond A. Frizzell,Melissa A. Ashlock,Paul A. Negulescu +20 more
TL;DR: In vitro pharmacology of VX-770, an orally bioavailable CFTR potentiator in clinical development for the treatment of CF, supports the hypothesis that pharmacological agents that restore or increase CFTR function can rescue epithelial cell function in human CF airway.
Journal ArticleDOI
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809
Fredrick Van Goor,Sabine Hadida,Peter D. J. Grootenhuis,B. Burton,Jeffrey H. Stack,Kimberly Straley,Caroline Decker,Mark R. Miller,Mccartney Jason,Eric R. Olson,Jeffrey J. Wine,Raymond A. Frizzell,Melissa A. Ashlock,Paul A. Negulescu +13 more
TL;DR: F508del-CFTR corrected by VX-809 exhibited biochemical and functional characteristics similar to normal CFTR, including biochemical susceptibility to proteolysis, residence time in the plasma membrane, and single-channel open probability.
Journal ArticleDOI
Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
Fredrick Van Goor,Kimberly Straley,Dong Cao,Jesus E. Gonzalez,Sabine Hadida,Anna R. Hazlewood,John Joubran,Tom Knapp,Lewis R. Makings,Mark Miller,Timothy Neuberger,Eric R. Olson,Victor Panchenko,James A. Rader,Ashvani K. Singh,Jeffrey H. Stack,Roger D. Tung,Peter D. J. Grootenhuis,Paul A. Negulescu +18 more
TL;DR: Two classes of novel, potent small molecules identified from screening compound libraries that restore the function of DeltaF508-CFTR in both recombinant cells and cultures of human bronchial epithelia isolated from CF patients are described.
Journal ArticleDOI
Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene
Patrick R. Sosnay,Karen R. Siklosi,Fredrick Van Goor,Kyle Kaniecki,Kyle Kaniecki,Haihui Yu,Neeraj Sharma,Anabela S. Ramalho,Anabela S. Ramalho,Margarida D. Amaral,Margarida D. Amaral,Ruslan Dorfman,Julian Zielenski,David L. Masica,Rachel Karchin,Linda Millen,Philip Thomas,George P. Patrinos,Mary Corey,Michelle Huckaby Lewis,Johanna M. Rommens,Carlo Castellani,Christopher M. Penland,Garry R. Cutting +23 more
TL;DR: It is illustrated that sourcing data directly from well-phenotyped subjects can address the gap in the ability to interpret clinically relevant genomic variation in patients with cystic fibrosis.
Journal ArticleDOI
VX-445-Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles.
Dominic Keating,Gautham Marigowda,Lucy D. Burr,Cori L. Daines,Marcus A. Mall,Edward F. McKone,Bonnie W. Ramsey,Steven M. Rowe,Laura A. Sass,Elizabeth Tullis,Charlotte M. McKee,Samuel M. Moskowitz,Sarah Robertson,Jessica H. Savage,Christopher Simard,Fredrick Van Goor,David Waltz,Fengjuan Xuan,Young Tomothy John,Jennifer L. Taylor-Cousar +19 more
TL;DR: The use of VX‐445–tezacaftor–ivacaftor to target Phe508del CFTR protein resulted in increased CFTR function in vitro and translated to improvements in patients with cystic fibrosis with one or two Phe48del alleles.