R
Ronald C. Rubenstein
Researcher at Children's Hospital of Philadelphia
Publications - 103
Citations - 6411
Ronald C. Rubenstein is an academic researcher from Children's Hospital of Philadelphia. The author has contributed to research in topics: Cystic fibrosis & Cystic fibrosis transmembrane conductance regulator. The author has an hindex of 36, co-authored 94 publications receiving 5675 citations. Previous affiliations of Ronald C. Rubenstein include Johns Hopkins University & University of Texas Southwestern Medical Center.
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Journal ArticleDOI
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
Frank J. Accurso,Steven M. Rowe,John P. Clancy,Michael P. Boyle,Jordan M. Dunitz,Peter R. Durie,Scott D. Sagel,Douglas B. Hornick,Michael W. Konstan,Scott H. Donaldson,Richard B. Moss,Joseph M. Pilewski,Ronald C. Rubenstein,Ahmet Uluer,Moira L. Aitken,Steven D. Freedman,Lynn M. Rose,Nicole Mayer-Hamblett,Qunming Dong,Jiuhong Zha,Anne Stone,Eric R. Olson,Claudia L. Ordoñez,Preston W. Campbell,Melissa A. Ashlock,Bonnie W. Ramsey +25 more
TL;DR: This study showed that VX-770 was associated with within-subject improvements in CFTR and lung function and provides support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis.
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Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
John P. Clancy,Steven M. Rowe,Frank J. Accurso,Moira L. Aitken,Raouf S. Amin,Melissa A. Ashlock,Manfred Ballmann,Michael P. Boyle,Inez Bronsveld,Preston W. Campbell,Kris De Boeck,Scott H. Donaldson,Henry L. Dorkin,Jordan M. Dunitz,Peter R. Durie,Manu Jain,Anissa Leonard,Karen McCoy,Richard B. Moss,Joseph M. Pilewski,Daniel B. Rosenbluth,Ronald C. Rubenstein,Michael S. Schechter,Martyn Botfield,Claudia L. Ordoñez,George T. Spencer-Green,Laurent Vernillet,Steve Wisseh,Karl Yen,Michael W. Konstan +29 more
TL;DR: In this study, VX-809 had a similar adverse event profile to placebo for 28 days in F508del-CFTR homozygous patients, and demonstrated biological activity with positive impact on CFTR function in the sweat gland.
Journal ArticleDOI
In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR.
TL;DR: 4PBA is a promising pharmacologic agent for inducing correction of the CF phenotype in CF patients carrying the delta F508 mutation, as detected by immunoblotting of whole cell lysates with anti-CFTR antisera.
Journal ArticleDOI
A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function.
TL;DR: Rubenstein et al. as mentioned in this paper presented data suggesting that 4PBA, at clinically achievable concentrations, induces CFTR channel function on the plasma membrane of deltaF508-expressing cystic fibrosis (CF) airway epithelial cells.
Journal ArticleDOI
Sodium 4-phenylbutyrate downregulates Hsc70: implications for intracellular trafficking of ΔF508-CFTR
TL;DR: Sodium 4-phenylbutyrate (4PBA) improves DeltaF508-CFTR trafficking and function in vitro in cystic fibrosis epithelial cells and in vivo, and data suggest that 4PBA may improve Deltaf508- CFTR trafficking by allowing a greater proportion of mutant CFTR to escape association with Hsc70.