Ronald Rubenstein, M.D., Ph.D.  rubenstein@wustl.edu

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Division Chief, Allergy, Immunology and Pulmonary Medicine
Professor of Pediatrics, Allergy and Pulmonary Medicine
Allergy, Immunology and Pulmonary Medicine

phone: (314) 454-2694

Clinical Interests

Dr. Rubenstein is a Professor of Pediatrics and Chief of the Division of Allergy and Pulmonary Medicine, having moved to Washington University in St. Louis in 2020. He received his undergraduate degree in chemistry from The Massachusetts Institute of Technology and medical degree and PhD in pharmacology from the University of Texas Southwestern Medical Center at Dallas. He subsequently completed a residency in Pediatrics at the Children’s Hospital of Pittsburgh and a Fellowship in Pediatric Pulmonology at the Johns Hopkins School of Medicine, where he became an Instructor in Pediatrics. He then moved to the Children’s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania where he Directed the Cystic Fibrosis Center, held the Richard B. Johnston, Jr. Endowed Chair in Pediatrics, and rose to the rank of Professor of Pediatrics with tenure. Dr. Rubenstein’s research is focused on understanding the mechanisms by which small molecules can repair the dysfunction of the most common Cystic Fibrosis-causing CFTR mutation, F508del. Through this work, he has become increasingly interested in the role of a novel chaperone of the endoplasmic reticulum, ERp29, in the biogenesis of proteins relevant to Cystic Fibrosis (CF) and other pulmonary diseases. This work has been supported by grants from the NIH, the Cystic Fibrosis Foundation including a LeRoy Matthews Physician Scientist Award, and an Established Investigator Award from the American Heart Association. He has also developed NIH- and CF Foundation-funded collaborative clinical and translational research projects focusing on CF-related diabetes, ototoxicity of aminoglycoside antibiotics in CF, and animal assisted therapy in the hospital setting, as well as participated in numerous multicenter clinical trials leading to the approval of novel therapies for CF. Dr. Rubenstein is a recognized expert in the care of people with Cystic Fibrosis, and serves as Chair of the CF Foundation Clinical Research Committee as well as a member of the CF Foundation Medical Advisory Council.

Education

  • BS, Massachusetts Institute of Technology1984
  • PhD, University of Texas Southwestern Graduate School of the Biomedical Sciences1990
  • MD, University of Texas Southwestern Medical School1991

Training

  • Residency in Pediatrics, The Children's Hospital of Pittsburgh1991 - 1994
  • Fellow in Pediatric Pulmonary Diseases, The Johns Hopkins School of Medicine1994 - 1997

Licensure and Board Certification

  • 1993 - PresMedical License, State of Pennsylvania
  • 1994 - 2008American Board of Pediatrics, General Pediatrics
  • 1994 - 2005Medical License, State of Maryland
  • 1998 - PresAmerican Board of Pediatrics, Pediatric Pulmonology
  • 2020 - PresMedical License, State of Missouri (pending)

Honors and Awards

  • Texaco Philanthropic Foundation Scholarship for Academic Achievement in Chemistry, Massachusetts Institute of Technology1983 - 1984
  • Merck Index Award for Outstanding Scholastic Achievement in Chemistry, Massachusetts Institute of Technology1984
  • Associate Member, Sigma Xi1984 - 1992
  • Alfred Gilman Memorial Award for Excellence in Graduate Research, Department of Pharmacology, University of Texas Southwestern Medical Center at Dallas1989
  • Member, Alpha Omega Alpha1990 - Pres
  • Member, Sigma Xi1992 - Pres
  • Leroy Matthews Physician Scientist Career Development Award, Cystic Fibrosis Foundation1996 - 2000
  • Helen B. Taussig Award for Post-doctoral Clinical Research, The Johns Hopkins School of Medicine1997
  • Established Investigator of the American Heart Association2002 - 2006
  • Member, Society for Pediatric Research2003 - Pres
  • The Children’s Hospital of Philadelphia Faculty Honor Roll2003 - 2015
  • “Faculty Teacher of the Year.” Presented by the Pediatrics Housestaff of The Children's Hospital of Philadelphia.2006
  • Dr. Milton Graub Medical Recognition Award, Delaware Valley Chapter, Cystic Fibrosis Foundation2018

Recent Publications view all (73)


Publication Co-Authors

  1. The Probable, Possible, and Novel Functions of ERp29. Front Physiol. 2020;1134. DOI=10.3389/fphys.2020.574339 . 
  2. Hypoglycemia and Islet Dysfunction following Oral Glucose Tolerance Testing in Pancreatic Insufficient Cystic Fibrosis. J Clin Endocrinol Metab. 2020. PMID:32668452 
  3. ERp29 as a regulator of Insulin biosynthesis. PLoS One. 2020;15(5):e0233502. PMCID:PMC7239452  PMID:32433667 
  4. Genetic potential and height velocity during childhood and adolescence do not fully account for shorter stature in cystic fibrosis. Pediatr Res. 2020. PMID:32386398 
  5. MON-660 Hypoglycemia Following OGTT Is More Frequent and Pronounced in CF Compared with Controls. J Endocr Soc. 2020;4:(Suppl 1):MON-660. doi:10.1210/jendso/bvaa046.458 . PMCID:PMC7208859  
  6. The EXTrauterine Environment for Neonatal Development Supports Normal Intestinal Maturation and Development. Cell Mol Gastroenterol Hepatol. 2020;10(3):623-637. doi: 10.1016/j.jcmgh.2020.05.006. Epub 2020 May 28. PMID:32474164 
  7. Dysregulated insulin in pancreatic insufficient cystic fibrosis with post-prandial hypoglycemia. J Cyst Fibros. 2020;19(2):310-315. PMCID:PMC7007375  PMID:31402215 
  8. Abnormal glucose tolerance and the 50-gram glucose challenge test in Cystic fibrosis. J Cyst Fibros. 2020. PMID:31974039 
  9. Risks associated with animal-assisted intervention programs: A literature review. Complement Ther Clin Pract. 2020;39:101145. 
  10. The presence of Aspergillus fumigatus is associated with worse respiratory quality of life in cystic fibrosis. J Cyst Fibros. 2020;19(1):125-130. PMID:31446018 
  11. The KDEL receptor has a role in the biogenesis and trafficking of the epithelial sodium channel (ENaC). J Biol Chem. 2019;294(48):18324-18336. PMCID:PMC6885628  PMID:31653700 
  12. Safety and efficacy of treatment with lumacaftor in combination with ivacaftor in younger patients with cystic fibrosis Expert Rev Respir Med. 2019;13:417-423. PMCID:PMC6482058  PMID:30929526 
  13. Islet Hormone and Incretin Secretion in Cystic Fibrosis after Four Months of Ivacaftor Therapy. Am J Respir Crit Care Med. 2019;199(3):342-351. PMCID:PMC6835076  PMID:30130412 
  14. β-Cell secretory defects are present in pancreatic insufficient cystic fibrosis with 1-hour oral glucose tolerance test glucose ≥155 mg/dL. Pediatr Diabetes. 2018;19(7):1173-1182. PMCID:PMC6364976  PMID:29885044 
  15. Editorial Focus: CFTR-dependent bicarbonate secretion by Calu-3 cells. Physiol Rep. 2018;6(e):13691. 
  16. Sexual and reproductive health care utilization and preferences reported by young women with cystic fibrosis. J Cyst Fibros. 2018;17(1):64-70. PMID:28866451 
  17. Sexual and reproductive health behaviors and experiences reported by young women with cystic fibrosis. J Cyst Fibros. 2018;17(1):57-63. PMID:28774749 
  18. Reduced β-Cell Secretory Capacity in Pancreatic-Insufficient, but Not Pancreatic-Sufficient, Cystic Fibrosis Despite Normal Glucose Tolerance. Diabetes. 2017;66(1):134-144. PMCID:PMC5204312  PMID:27495225 
  19. Elevation of one hour plasma glucose during oral glucose tolerance testing. Pediatr Pulmonol. 2015;50(10):963-9. PMCID:PMC6910238  PMID:26087115 
  20. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015;3(7):524-33. PMCID:PMC4641035  PMID:26070913 
  21. Long-term treatment with oral N-acetylcysteine: affects lung function but not sputum inflammation in cystic fibrosis subjects. A phase II randomized placebo-controlled trial. J Cyst Fibros. 2015;14(2):219-27. PMID:25228446 
  22. ERp29 regulates epithelial sodium channel functional expression by promoting channel cleavage. Am J Physiol Cell Physiol. 2014;307(8):C701-9. PMCID:PMC4199997  PMID:24944201 
  23. Patient and parent perceptions of the diagnosis and management of cystic fibrosis-related diabetes. J Clin Transl Endocrinol. 2014;1(3):100-107. PMCID:PMC5685035  PMID:29159090 
  24. IL-17A induces Pendrin expression and chloride-bicarbonate exchange in human bronchial epithelial cells. PLoS One. 2014;9(8):e103263. PMCID:PMC4139276  PMID:25141009 
  25. Body composition and pulmonary function in cystic fibrosis. Front Pediatr. 2014;2:33. PMCID:PMC3995066  PMID:24783186 
  26. Seeing is believing: Imaging early lung disease in cystic fibrosis. Am J Respir Crit Care Med. 2014;189:883-5. 
  27. On preventing the extinction of the physician-scientist in pediatric pulmonology Front Pediatr. 2014;2(4):1-6. PMCID:PMC3896875  PMID:24479109 
  28. Hsc70 negatively regulates epithelial sodium channel trafficking at multiple sites in epithelial cells. Am J Physiol Cell Physiol. 2013;305(7):C776-87. PMCID:PMC3798670  PMID:23885065 
  29. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013;68(9):818-25. PMCID:PMC3756431  PMID:23749840 
  30. High prevalence of aquagenic wrinkling of the palms in patients with cystic fibrosis and association with measurable increases in transepidermal water loss. Pediatr Dermatol. 2012;29(5):560-6. PMID:22471628 
  31. Molecular Chaperones as Targets to Circumvent the CFTR Defect in Cystic Fibrosis. Front Pharmacol. 2012;3:137. PMCID:PMC3398409  PMID:22822398 
  32. Cystic fibrosis transmembrane conductance regulator contributes to reacidification of alkalinized lysosomes in RPE cells. Am J Physiol Cell Physiol. 2012;303(2):C160-9. PMCID:PMC3404519  PMID:22572847 
  33. Multiple residues in the distal C terminus of the α-subunit have roles in modulating human epithelial sodium channel activity. Am J Physiol Renal Physiol. 2012;303(2):F220-8. PMCID:PMC3404586  PMID:22573385 
  34. Hsp70 promotes epithelial sodium channel functional expression by increasing its association with coat complex II and its exit from endoplasmic reticulum. J Biol Chem. 2012;287(23):19255-65. PMCID:PMC3365957  PMID:22496374 
  35. Future Directions in Early Cystic Fibrosis Lung Disease Research: an NHLBI workshop report. Am J Respir Crit Care Med. 2012;185:887-892. 
  36. 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. Thorax. 2012;67(1):12-8. PMCID:PMC3746507  PMID:21825083 
  37. 4-Phenylbutyrate stimulates Hsp70 expression through the Elp2 component of elongator and STAT-3 in cystic fibrosis epithelial cells. J Biol Chem. 2011;286(52):45083-92. PMCID:PMC3247989  PMID:22069317 
  38. Nocturnal saturation and glucose tolerance in children with cystic fibrosis. Thorax. 2011;66(7):574-8. PMID:21273357 
  39. ERp29 regulates DeltaF508 and wild-type cystic fibrosis transmembrane conductance regulator (CFTR) trafficking to the plasma membrane in cystic fibrosis (CF) and non-CF epithelial cells. J Biol Chem. 2011;286(24):21239-53. PMCID:PMC3122184  PMID:21525008 
  40. Elevation of 1-hour plasma glucose during oral glucose tolerance testing is associated with worse pulmonary function in cystic fibrosis. Diabetes Care. 2011;34(2):292-5. PMCID:PMC3024336  PMID:21228248 
  41. Regulation of endogenous ENaC functional expression by CFTR and ΔF508-CFTR in airway epithelial cells. Am J Physiol Lung Cell Mol Physiol. 2011;300(1):L88-L101. PMCID:PMC3023291  PMID:20935229 
  42. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010;363(21):1991-2003. PMCID:PMC3148255  PMID:21083385 
  43. ERp29 restricts Connexin43 oligomerization in the endoplasmic reticulum. Mol Biol Cell. 2009;20(10):2593-604. PMCID:PMC2682600  PMID:19321666 
  44. Carrier Screening, Incidence of Cystic Fibrosis and Difficult Decisions. J Amer Med Assoc. 2009;302:2595-6. 
  45. Small heat shock protein alphaA-crystallin regulates epithelial sodium channel expression. J Biol Chem. 2007;282(38):28149-56. PMCID:PMC2361386  PMID:17664274 
  46. Intracellular trafficking of a polymorphism in the COOH terminus of the alpha-subunit of the human epithelial sodium channel is modulated by casein kinase 1. Am J Physiol Renal Physiol. 2007;293(3):F868-76. PMID:17596527 
  47. Murine nasal septa for respiratory epithelial air-liquid interface cultures. Biotechniques. 2007;43(2):195-6, 198, 200 passim. PMID:17824387 
  48. Regulatory interactions of N1303K-CFTR and ENaC in Xenopus oocytes: evidence that chloride transport is not necessary for inhibition of ENaC. Am J Physiol Cell Physiol. 2007;292(4):C1553-61. PMID:17182731 
  49. Abnormal regulatory interactions of I148T-CFTR and the epithelial Na+ channel in Xenopus oocytes. Am J Physiol Cell Physiol. 2007;292(1):C603-11. PMID:16822950 
  50. Cystic fibrosis as a risk factor for recurrent venous thrombosis at a pediatric tertiary care hospital. J Pediatr. 2006;148(5):659-64. PMID:16737881 
  51. Differential effects of Hsc70 and Hsp70 on the intracellular trafficking and functional expression of epithelial sodium channels. Proc Natl Acad Sci U S A. 2006;103(15):5817-22. PMCID:PMC1458656  PMID:16585520 
  52. Risk factors for death of patients with cystic fibrosis awaiting lung transplantation. Am J Respir Crit Care Med. 2006;173(6):659-66. PMCID:PMC2662949  PMID:16387803 
  53. Differential modulation of a polymorphism in the COOH terminus of the alpha-subunit of the human epithelial sodium channel by protein kinase Cdelta. Am J Physiol Renal Physiol. 2006;290(2):F279-88. PMID:16174865 
  54. Targeted therapy for cystic fibrosis: cystic fibrosis transmembrane conductance regulator mutation-specific pharmacologic strategies. Mol Diagn Ther. 2006;10(5):293-301. PMID:17022692 
  55. Novel, mechanism-based therapies for cystic fibrosis. Curr Opin Pediatr. 2005;17(3):385-92. PMID:15891431 
  56. Functional polymorphism in the carboxyl terminus of the alpha-subunit of the human epithelial sodium channel. J Biol Chem. 2004;279(23):23900-7. PMID:15069064 
  57. Cystic fibrosis transmembrane conductance regulator differentially regulates human and mouse epithelial sodium channels in Xenopus oocytes. J Biol Chem. 2004;279(22):23183-92. PMID:15047694 
  58. Expert Panelist Opinion regarding consideration of research under HHS regulations at 45 CFR 46.407. Office for Human Research Protections, Department of Health and Human Services. United States Government.Published at http://ohrp.osophs.dhhs.gov/panels/407-02pnl/exp1.pdf 2003. 
  59. Genistein improves regulatory interactions between G551D-cystic fibrosis transmembrane conductance regulator and the epithelial sodium channel in Xenopus oocytes. J Biol Chem. 2002;277(52):50341-7. PMID:12386156 
  60. Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate. Mol Ther. 2002;6(1):119-26. PMID:12095312 
  61. Genistein restores functional interactions between Delta F508-CFTR and ENaC in Xenopus oocytes. J Biol Chem. 2002;277(11):8928-33. PMID:11773060 
  62. Sodium 4-phenylbutyrate downregulates HSC70 expression by facilitating mRNA degradation. Am J Physiol Lung Cell Mol Physiol. 2001;281(1):L43-51. PMID:11404244 
  63. A simplified cyclic adenosine monophosphate-mediated sweat rate test for quantitative measure of cystic fibrosis transmembrane regulator (CFTR) function. J Pediatr. 2000;137(6):849-55. PMID:11113843 
  64. Mutation in the gene responsible for cystic fibrosis and predisposition to chronic rhinosinusitis in the general population. JAMA. 2000;284(14):1814-9. PMID:11025834 
  65. Sodium 4-phenylbutyrate downregulates Hsc70: implications for intracellular trafficking of DeltaF508-CFTR. Am J Physiol Cell Physiol. 2000;278(2):C259-67. PMID:10666020 
  66. CFTR is functionally active in GnRH-expressing GT1-7 hypothalamic neurons. Am J Physiol. 1999;277(3):C563-71. PMID:10484343 
  67. Use of protein repair therapy in the treatment of cystic fibrosis. Curr Opin Pediatr. 1998;10(3):250-5. PMID:9716885 
  68. A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function. Am J Respir Crit Care Med. 1998;157(2):484-90. PMID:9476862 
  69. In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR. J Clin Invest. 1997;100(10):2457-65. PMCID:PMC508446  PMID:9366560 
  70. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther. 1997;4(5):384-92. PMID:9274714 
  71. Acute flank pain and reversible renal dysfunction associated with nonsteroidal anti-inflammatory drug use. Pediatrics. 1993;92(3):459-60. PMID:8361805 
  72. Selectivity of the beta-adrenergic receptor among Gs, Gi's, and Go: assay using recombinant alpha subunits in reconstituted phospholipid vesicles. Biochemistry. 1991;30(44):10769-77. PMID:1657154 
  73. The hydrophobic tryptic core of the beta-adrenergic receptor retains Gs regulatory activity in response to agonists and thiols. J Biol Chem. 1987;262(34):16655-62. PMID:2890639 
Last updated: 09/18/2020
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