Thomas Ferkol, M.D.  ferkol_t@wustl.edu

Director, Division of Allergy, Immunology, and Pulmonary Medicine
Professor of Cell Biology and Physiology
Alexis Hartmann Professor of Pediatrics
Researcher, Pathobiology
Cell Biology & PhysiologyAllergy, Immunology and Pulmonary MedicinePathobiology

phone: (314) 454-2694

Clinical Interests

Thomas Ferkol, MD is the Alexis Hartmann Professor of Pediatrics, Professor of Cell Biology and Physiology, and Director of the multidisciplinary Division of Pediatric Allergy, Immunology, and Pulmonary Medicine at the Washington University School of Medicine. Dr. Ferkol graduated from Case Western Reserve University (Cleveland, Ohio) in 1981, and he received his M.D. degree from the Ohio State University College of Medicine (Columbus, Ohio) in 1985. He was a pediatric resident at the University of North Carolina at Chapel Hill (1985-1988), where he also served as Chief Resident and Clinical Instructor (1988-1989). Dr. Ferkol returned to Case Western Reserve University in 1989 for fellowship training in pediatric pulmonology, and subsequently joined the faculty of Case Western Reserve University and Rainbow Babies and Children’s Hospital. Dr. Ferkol moved to Washington University School of Medicine in 2000, where he is currently Director of the Division of Pediatric Allergy, Immunology, and Pulmonary Medicine. He is the past director of the comprehensive Washington University Cystic Fibrosis Center, and directed premier clinical and research programs in cystic fibrosis. The Center is a member of the Cystic Fibrosis Foundation Therapeutics Development Network. An American Lung Association Edward Livingston Trudeau Scholar and recipient of the Cystic Fibrosis Foundation (CFF) LeRoy Matthew’s Physician-Scientist Award, his research has focused on characterizing genetic and molecular factors that contribute to lung disease in cystic fibrosis and primary ciliary dyskinesia. With past and continued support from the National Institutes of Health (NIH), CFF, and March of Dimes, Dr. Ferkol and his collaborators are studying the origins and potential therapeutic targets for these inherited airway diseases. He is an investigator for the NIH-supported Genetic Diseases of Mucociliary Clearance Consortium, and has assembled clinical, biomedical, and engineering research collaborative at Washington University, studying the genetic and phenotypic spectrum of ciliopathies. He is also co-principal investigator on a NIH-funded program that is examining adverse respiratory outcomes in children with chronic lung disease related to prematurity. Dr. Ferkol has been an active member of the American Thoracic Society (ATS), serving as the chair of the Scientific Assembly on Pediatrics and chair of its program committee. He was recently installed as president of the ATS, only the second pediatrician in the 110 years of the Society. He has been a member of several scientific review groups, including the NIH Gene and Drug Delivery Study Section, ATS Career Development Grants Review Committee, CFF Research Development Program Review Committee and CFF Functional Genomics Review Committee. Finally, he serves as a member of the American Board of Pediatrics Subboard of Pediatric Pulmonology.

Education

  • B.A., magna cum laude, Case Western Reserve University1981
  • MB, University of Nottingham, School of Biological Sciences1980
  • M.D., Medicinae doctor, The Ohio State University College of Medicine1985

Training

  • Intern, University of North Carolina at Chapel Hill1985 - 1986
  • Resident, University of North Carolina at Chapel Hill1986 - 1988
  • Chief Resident, University of North Carolina at Chapel Hill1988 - 1989
  • Pulmonology Fellow, Case Western Reserve University School of Medicine1989 - 1992

Licensure and Board Certification

  • Medical License, State of Missouri (current)
  • Medical License, State of North Carolina (expired voluntarily)
  • Medical License, State of Ohio (expired voluntarily)
  • National Board of Medical Examiners, Diplomate
  • US Drug Enforcement Agency, Certified
  • American Board of Pediatrics, General Pediatrics Diplomate, Certified (Re-certified, 1998, 2005)1990
  • American Board of Pediatrics, Pediatric Pulmonology Subboard Diplomate (Re-certified, 2002, 2009)1994

Honors

  • Cystic Fibrosis Foundation LeRoy Matthews Physician-Scientist Award1989
  • Floyd Denny Pediatric Alumni Society Award, Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina1989
  • Rainbow Babies and Children’s Hospital Trustees New Investigator Award1992
  • American Lung Association Edward Livingston Trudeau Scholar1994
  • Teaching Excellence Award, Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, Ohio1994
  • Society for Pediatric Research, member1997
  • Best Doctors of America®1998 - Pres
  • EDI Innovation Award.1999
  • European Cystic Fibrosis Conference Presentation Award2004
  • America's Top Pediatricians, Consumer's Research Council of America2006 - Pres
  • American Pediatric Society, member2009 - Pres
  • Alexis Hartmann MD Professorship Award2012

Recent Publications view all (133)


  1. Leigh MW, Ferkol TW, Davis SD, Lee HS, Rosenfeld M, Dell SD, Sagel SD, Milla C, Olivier KN, Sullivan KM, Zariwala MA, Pittman JE, Shapiro AJ, Carson JL, Krischer J, Hazucha MJ, Knowles MR. Clinical Features and Associated Likelihood of Primary Ciliary Dyskinesia in Children and Adolescents. Ann Am Thorac Soc. 2016;13(8):1305-13. PMID:27070726  
  2. Dell SD, Leigh MW, Lucas JS, Ferkol TW, Knowles MR, Alpern A, Behan L, Morris AM, Hogg C, DunnGalvin A, Quittner AL. Primary Ciliary Dyskinesia: First Health-related Quality of Life Measures for Pediatric Patients. Ann Am Thorac Soc. 2016. PMID:27464304  
  3. Horani A, Ferkol TW. Primary ciliary dyskinesia and associated sensory ciliopathies. Expert Rev Respir Med. 2016;10(5):569-76. PMCID:PMC4893162  PMID:26967669  
  4. Sterni LM, Collaco JM, Baker CD, Carroll JL, Sharma GD, Brozek JL, Finder JD, Ackerman VL, Arens R, Boroughs DS, Carter J, Daigle KL, Dougherty J, Gozal D, Kevill K, Kravitz RM, Kriseman T, MacLusky I, Rivera-Spoljaric K, Tori AJ, Ferkol T, Halbower AC, ATS Pediatric Chronic Home Ventilation Workgroup. An Official American Thoracic Society Clinical Practice Guideline: Pediatric Chronic Home Invasive Ventilation. Am J Respir Crit Care Med. 2016;193(8):e16-35. PMID:27082538  
  5. Horani A, Ferkol TW, Dutcher SK, Brody SL. Genetics and biology of primary ciliary dyskinesia. Paediatr Respir Rev. 2016;18:18-24. PMCID:PMC4864047  PMID:26476603  
  6. Morgan WJ, VanDevanter DR, Pasta DJ, Foreman AJ, Wagener JS, Konstan MW, Scientific Advisory Group, Investigators and Coordinators of Epidemiologic Study of Cystic Fibrosis. Forced Expiratory Volume in 1 Second Variability Helps Identify Patients with Cystic Fibrosis at Risk of Greater Loss of Lung Function. J Pediatr. 2016;169:116-21.e2. PMID:26388208  
  7. Shapiro AJ, Zariwala MA, Ferkol T, Davis SD, Sagel SD, Dell SD, Rosenfeld M, Olivier KN, Milla C, Daniel SJ, Kimple AJ, Manion M, Knowles MR, Leigh MW, Genetic Disorders of Mucociliary Clearance Consortium. Diagnosis, monitoring, and treatment of primary ciliary dyskinesia: PCD foundation consensus recommendations based on state of the art review. Pediatr Pulmonol. 2016;51(2):115-32. PMCID:PMC4912005  PMID:26418604  
  8. Zar HJ, Billo N, Ferkol T, Vestbo J. Decade of the lung--a call for action to promote lung health globally. Lancet Respir Med. 2016;4(1):e3-4. PMID:26762667  
  9. Poindexter BB, Feng R, Schmidt B, Aschner JL, Ballard RA, Hamvas A, Reynolds AM, Shaw PA, Jobe AH, Prematurity and Respiratory Outcomes Program. Comparisons and Limitations of Current Definitions of Bronchopulmonary Dysplasia for the Prematurity and Respiratory Outcomes Program. Ann Am Thorac Soc. 2015;12(12):1822-30. PMCID:PMC4722827  PMID:26397992  
  10. Ferkol T, Quinton P. Precision Medicine: At What Price? Am J Respir Crit Care Med. 2015;192(6):658-9. PMID:26207804  
  11. Heltshe SL, Saiman L, Popowitch EB, Miller MB, Kloster M, Thompson V, Ferkol TW, Hoover WC, Schechter MS, Muhlebach MS. Outcomes and Treatment of Chronic Methicillin-Resistant Staphylococcus aureus Differs by Staphylococcal Cassette Chromosome mec (SCCmec) Type in Children With Cystic Fibrosis. J Pediatric Infect Dis Soc. 2015;4(3):225-31. PMCID:PMC4554198  PMID:26336603  
  12. Cogen J, Emerson J, Sanders DB, Ren C, Schechter MS, Gibson RL, Morgan W, Rosenfeld M, EPIC Study Group. Risk factors for lung function decline in a large cohort of young cystic fibrosis patients. Pediatr Pulmonol. 2015;50(8):763-70. PMID:26061914  
  13. Sanders DB, Emerson J, Ren CL, Schechter MS, Gibson RL, Morgan W, Rosenfeld M, EPIC Study Group. Early Childhood Risk Factors for Decreased FEV1 at Age Six to Seven Years in Young Children with Cystic Fibrosis. Ann Am Thorac Soc. 2015;12(8):1170-6. PMCID:PMC4566413  PMID:26288390  
  14. Maitre NL, Ballard RA, Ellenberg JH, Davis SD, Greenberg JM, Hamvas A, Pryhuber GS, Prematurity and Respiratory Outcomes Program. Respiratory consequences of prematurity: evolution of a diagnosis and development of a comprehensive approach. J Perinatol. 2015;35(5):313-21. doi:10.1038/jp.2015.19  PMCID:PMC4414744  PMID:25811285  
  15. Coste F, Ferkol T, Hamvas A, Cleveland C, Linneman L, Hoffman J, Kemp J. Ventilatory control and supplemental oxygen in premature infants with apparent chronic lung disease. Arch Dis Child Fetal Neonatal Ed. 2015;100(3):F233-7. doi:10.1136/archdischild-2014-307272  PMID:25716677  
  16. Faro A, Wood RE, Schechter MS, Leong AB, Wittkugel E, Abode K, Chmiel JF, Daines C, Davis S, Eber E, Huddleston C, Kilbaugh T, Kurland G, Midulla F, Molter D, Montgomery GS, Retsch-Bogart G, Rutter MJ, Visner G, Walczak SA, Ferkol TW, Michelson PH, American Thoracic Society Ad Hoc Committee on Flexible Airway Endoscopy in Children. Official American Thoracic Society technical standards: flexible airway endoscopy in children. Am J Respir Crit Care Med. 2015;191(9):1066-80. doi:10.1164/rccm.201503-0474ST  PMID:25932763  
  17. Pryhuber GS, Maitre NL, Ballard RA, Cifelli D, Davis SD, Ellenberg JH, Greenberg JM, Kemp J, Mariani TJ, Panitch H, Ren C, Shaw P, Taussig LM, Hamvas A, Prematurity and Respiratory Outcomes Program Investigators. Prematurity and respiratory outcomes program (PROP): study protocol of a prospective multicenter study of respiratory outcomes of preterm infants in the United States. BMC Pediatr. 2015;15(1):37. doi:10.1186/s12887-015-0346-3  PMCID:PMC4407843  PMID:25886363  
  18. Towe C, Chester Ogborn A, Ferkol T, Sweet S, Huddleston C, White F, Faro A. Bronchiolitis obliterans syndrome is not specific for bronchiolitis obliterans in pediatric lung transplant. J Heart Lung Transplant. 2015;34(4):516-21. doi:10.1016/j.healun.2014.10.004  PMID:25499141  
  19. Pittman JE, Ferkol TW. The Evolution of Cystic Fibrosis Care. Chest. 2015. doi:10.1378/chest.14-1997  PMID:25764168  
  20. Muhlebach MS, Heltshe SL, Popowitch EB, Miller MB, Thompson V, Kloster M, Ferkol T, Hoover WC, Schechter MS, Saiman L, STAR-CF Study Team. Multicenter Observational Study on Factors and Outcomes Associated with Various Methicillin-Resistant Staphylococcus aureus Types in Children with Cystic Fibrosis. Ann Am Thorac Soc. 2015. doi:10.1513/AnnalsATS.201412-596OC  PMID:25745825  
  21. Davis SD, Ferkol TW, Rosenfeld M, Lee HS, Dell SD, Sagel SD, Milla C, Zariwala MA, Pittman JE, Shapiro AJ, Carson JL, Krischer JP, Hazucha MJ, Cooper ML, Knowles MR, Leigh MW. Clinical features of childhood primary ciliary dyskinesia by genotype and ultrastructural phenotype. Am J Respir Crit Care Med. 2015;191(3):316-24. doi:10.1164/rccm.201409-1672OC  PMCID:PMC4351577  PMID:25493340  
  22. Zemanick ET, Emerson J, Thompson V, McNamara S, Morgan W, Gibson RL, Rosenfeld M, EPIC Study Group. Clinical outcomes after initial pseudomonas acquisition in cystic fibrosis. Pediatr Pulmonol. 2015;50(1):42-8. doi:10.1002/ppul.23036  PMID:24644274  
  23. Daines C, VanDeVanter D, Khan U, Emerson J, Heltshe S, McNamara S, Anstead M, Langkamp M, Doring G, Ratjen F, Ramsey B, Gibson RL, Morgan W, Rosenfeld M, EPIC Investigators. Serology as a diagnostic tool for predicting initialPseudomonas aeruginosa acquisition in children with cystic fibrosis. J Cyst Fibros. 2014;13(5):542-9. doi:10.1016/j.jcf.2014.06.005  PMID:25027419  
  24. Ulm LN, Hamvas A, Ferkol TW, Rodriguez OM, Cleveland CM, Linneman LA, Hoffmann JA, Sicard-Su MJ, Kemp JS. Sources of methodological variability in phase angles from respiratory inductance plethysmography in preterm infants. Ann Am Thorac Soc. 2014;11(5):753-60. doi:10.1513/AnnalsATS.201310-363OC  PMID:24716708  
  25. Zar HJ, Ferkol TW. The global burden of respiratory disease-impact on child health. Pediatr Pulmonol. 2014;49(5):430-4. doi:10.1002/ppul.23030  PMID:24610581  
  26. Deady LE, Todd EM, Davis CG, Zhou JY, Topcagic N, Edelson BT, Ferkol TW, Cooper MA, Muenzer JT, Morley SC. L-plastin is essential for alveolar macrophage production and control of pulmonary pneumococcal infection. Infect Immun. 2014;82(5):1982-93. doi:10.1128/IAI.01199-13  PMCID:PMC3993441  PMID:24595139  
  27. Pittman JE, Cutting G, Davis SD, Ferkol T, Boucher R. Cystic fibrosis: NHLBI Workshop on the Primary Prevention of Chronic Lung Diseases. Ann Am Thorac Soc. 2014;11 Suppl 3:S161-8. doi:10.1513/AnnalsATS.201312-444LD  PMID:24754825  
  28. Knowles MR, Ostrowski LE, Leigh MW, Sears PR, Davis SD, Wolf WE, Hazucha MJ, Carson JL, Olivier KN, Sagel SD, Rosenfeld M, Ferkol TW, Dell SD, Milla CE, Randell SH, Yin W, Sannuti A, Metjian HM, Noone PG, Noone PJ, Olson CA, Patrone MV, Dang H, Lee HS, Hurd TW, Gee HY, Otto EA, Halbritter J, Kohl S, Kircher M, Krischer J, Bamshad MJ, Nickerson DA, Hildebrandt F, Shendure J, Zariwala MA. Mutations in RSPH1 cause primary ciliary dyskinesia with a unique clinical and ciliary phenotype. Am J Respir Crit Care Med. 2014;189(6):707-17. doi:10.1164/rccm.201311-2047OC  PMCID:PMC3983840  PMID:24568568  
  29. Ferkol T, Schraufnagel D. The global burden of respiratory disease. Ann Am Thorac Soc. 2014;11(3):404-6. doi:10.1513/AnnalsATS.201311-405PS  PMID:24673696  
  30. Shapiro AJ, Davis SD, Ferkol T, Dell SD, Rosenfeld M, Olivier KN, Sagel SD, Milla C, Zariwala MA, Wolf W, Carson JL, Hazucha MJ, Burns K, Robinson B, Knowles MR, Leigh MW. Laterality defects other than situs inversus totalis in primary ciliary dyskinesia: insights into situs ambiguus and heterotaxy. Chest. 2014. doi:10.1378/chest.13-1704  PMID:24577564  
  31. Horani A, Brody SL, Ferkol TW. Picking up speed: advances in the genetics of primary ciliary dyskinesia. Pediatr Res. 2014;75(1-2):158-64. doi:10.1038/pr.2013.200  PMCID:PMC3946436  PMID:24192704  
  32. Cohen-Cymberknoh M, Kerem E, Ferkol T, Elizur A. Airway inflammation in cystic fibrosis: molecular mechanisms and clinical implications. Thorax. 2013;68(12):1157-62. doi:10.1136/thoraxjnl-2013-203204  PMID:23704228  
  33. Leigh MW, Hazucha MJ, Chawla KK, Baker BR, Shapiro AJ, Brown DE, Lavange LM, Horton BJ, Qaqish B, Carson JL, Davis SD, Dell SD, Ferkol TW, Atkinson JJ, Olivier KN, Sagel SD, Rosenfeld M, Milla C, Lee HS, Krischer J, Zariwala MA, Knowles MR. Standardizing nasal nitric oxide measurement as a test for primary ciliary dyskinesia. Ann Am Thorac Soc. 2013;10(6):574-81. doi:10.1513/AnnalsATS.201305-110OC  PMCID:PMC3960971  PMID:24024753  
  34. Chen DL, Atkinson JJ, Ferkol TW. FDG PET imaging in cystic fibrosis. Semin Nucl Med. 2013;43(6):412-9. doi:10.1053/j.semnuclmed.2013.06.002  PMID:24094708  
  35. Knowles MR, Ostrowski LE, Loges NT, Hurd T, Leigh MW, Huang L, Wolf WE, Carson JL, Hazucha MJ, Yin W, Davis SD, Dell SD, Ferkol TW, Sagel SD, Olivier KN, Jahnke C, Olbrich H, Werner C, Raidt J, Wallmeier J, Pennekamp P, Dougherty GW, Hjeij R, Gee HY, Otto EA, Halbritter J, Chaki M, Diaz KA, Braun DA, Porath JD, Schueler M, Baktai G, Griese M, Turner EH, Lewis AP, Bamshad MJ, Nickerson DA, Hildebrandt F, Shendure J, Omran H, Zariwala MA. Mutations in SPAG1 cause primary ciliary dyskinesia associated with defective outer and inner dynein arms. Am J Hum Genet. 2013;93(4):711-20. doi:10.1016/j.ajhg.2013.07.025  PMCID:PMC3791252  PMID:24055112  
  36. Austin-Tse C, Halbritter J, Zariwala MA, Gilberti RM, Gee HY, Hellman N, Pathak N, Liu Y, Panizzi JR, Patel-King RS, Tritschler D, Bower R, O'Toole E, Porath JD, Hurd TW, Chaki M, Diaz KA, Kohl S, Lovric S, Hwang DY, Braun DA, Schueler M, Airik R, Otto EA, Leigh MW, Noone PG, Carson JL, Davis SD, Pittman JE, Ferkol TW, Atkinson JJ, Olivier KN, Sagel SD, Dell SD, Rosenfeld M, Milla CE, Loges NT, Omran H, Porter ME, King SM, Knowles MR, Drummond IA, Hildebrandt F. Zebrafish Ciliopathy Screen Plus Human Mutational Analysis Identifies C21orf59 and CCDC65 Defects as Causing Primary Ciliary Dyskinesia. Am J Hum Genet. 2013;93(4):672-86. doi:10.1016/j.ajhg.2013.08.015  PMCID:PMC3791264  PMID:24094744  
  37. Morgan WJ, Wagener JS, Yegin A, Pasta DJ, Millar SJ, Konstan MW, Scientific Advisory Group, investigators, coordinators of the Epidemiologic Study of Cystic Fibrosis. Probability of treatment following acute decline in lung function in children with cystic fibrosis is related to baseline pulmonary function. J Pediatr. 2013;163(4):1152-7.e2. doi:10.1016/j.jpeds.2013.05.013  PMCID:PMC4064589  PMID:23810128  
  38. Mayer-Hamblett N, Rosenfeld M, Treggiari MM, Konstan MW, Retsch-Bogart G, Morgan W, Wagener J, Gibson RL, Khan U, Emerson J, Thompson V, Elkin EP, Ramsey BW, EPIC, ESCF Investigators. Standard care versus protocol based therapy for new onset Pseudomonas aeruginosa in cystic fibrosis. Pediatr Pulmonol. 2013;48(10):943-53. doi:10.1002/ppul.22693  PMCID:PMC4059359  PMID:23818295  
  39. Horani A, Brody SL, Ferkol TW, Shoseyov D, Wasserman MG, Ta-shma A, Wilson KS, Bayly PV, Amirav I, Cohen-Cymberknoh M, Dutcher SK, Elpeleg O, Kerem E. CCDC65 mutation causes primary ciliary dyskinesia with normal ultrastructure and hyperkinetic cilia. PLoS One. 2013;8(8):e72299. doi:10.1371/journal.pone.0072299  PMCID:PMC3753302  PMID:23991085  
  40. Zariwala MA, Gee HY, Kurkowiak M, Al-Mutairi DA, Leigh MW, Hurd TW, Hjeij R, Dell SD, Chaki M, Dougherty GW, Adan M, Spear PC, Esteve-Rudd J, Loges NT, Rosenfeld M, Diaz KA, Olbrich H, Wolf WE, Sheridan E, Batten TF, Halbritter J, Porath JD, Kohl S, Lovric S, Hwang DY, Pittman JE, Burns KA, Ferkol TW, Sagel SD, Olivier KN, Morgan LC, Werner C, Raidt J, Pennekamp P, Sun Z, Zhou W, Airik R, Natarajan S, Allen SJ, Amirav I, Wieczorek D, Landwehr K, Nielsen K, Schwerk N, Sertic J, Köhler G, Washburn J, Levy S, Fan S, Koerner-Rettberg C, Amselem S, Williams DS, Mitchell BJ, Drummond IA, Otto EA, Omran H, Knowles MR, Hildebrandt F. ZMYND10 is mutated in primary ciliary dyskinesia and interacts with LRRC6. Am J Hum Genet. 2013;93(2):336-45. doi:10.1016/j.ajhg.2013.06.007  PMCID:PMC3738827  PMID:23891469  
  41. Ferkol TW, Puffenberger EG, Lie H, Helms C, Strauss KA, Bowcock A, Carson JL, Hazucha M, Morton DH, Patel AC, Leigh MW, Knowles MR, Zariwala MA. Primary ciliary dyskinesia-causing mutations in Amish and Mennonite communities. J Pediatr. 2013;163(2):383-7. doi:10.1016/j.jpeds.2013.01.061  PMCID:PMC3725203  PMID:23477994  
  42. Davis SD, Ferkol T. Identifying the origins of cystic fibrosis lung disease. N Engl J Med. 2013;368(21):2026-8. doi:10.1056/NEJMe1303487  PMID:23692172  
  43. Antony D, Becker-Heck A, Zariwala MA, Schmidts M, Onoufriadis A, Forouhan M, Wilson R, Taylor-Cox T, Dewar A, Jackson C, Goggin P, Loges NT, Olbrich H, Jaspers M, Jorissen M, Leigh MW, Wolf WE, Daniels ML, Noone PG, Ferkol TW, Sagel SD, Rosenfeld M, Rutman A, Dixit A, O'Callaghan C, Lucas JS, Hogg C, Scambler PJ, Emes RD, Uk10k, Chung EM, Shoemark A, Knowles MR, Omran H, Mitchison HM. Mutations in CCDC39 and CCDC40 are the major cause of primary ciliary dyskinesia with axonemal disorganization and absent inner dynein arms. Hum Mutat. 2013;34(3):462-72. doi:10.1002/humu.22261  PMCID:PMC3630464  PMID:23255504  
  44. Knowles MR, Leigh MW, Ostrowski LE, Huang L, Carson JL, Hazucha MJ, Yin W, Berg JS, Davis SD, Dell SD, Ferkol TW, Rosenfeld M, Sagel SD, Milla CE, Olivier KN, Turner EH, Lewis AP, Bamshad MJ, Nickerson DA, Shendure J, Zariwala MA, Genetic Disorders of Mucociliary Clearance Consortium. Exome sequencing identifies mutations in CCDC114 as a cause of primary ciliary dyskinesia. Am J Hum Genet. 2013;92(1):99-106. doi:10.1016/j.ajhg.2012.11.003  PMCID:PMC3542458  PMID:23261302  
  45. Horani A, Ferkol TW, Shoseyov D, Wasserman MG, Oren YS, Kerem B, Amirav I, Cohen-Cymberknoh M, Dutcher SK, Brody SL, Elpeleg O, Kerem E. LRRC6 mutation causes primary ciliary dyskinesia with dynein arm defects. PLoS One. 2013;8(3):e59436. doi:10.1371/journal.pone.0059436  PMCID:PMC3602302  PMID:23527195  
  46. Ross K, Chmiel JF, Ferkol T. The impact of the Clean Air Act. J Pediatr. 2012;161(5):781-6. doi:10.1016/j.jpeds.2012.06.064  PMID:22920509  
  47. Horani A, Druley TE, Zariwala MA, Patel AC, Levinson BT, Van Arendonk LG, Thornton KC, Giacalone JC, Albee AJ, Wilson KS, Turner EH, Nickerson DA, Shendure J, Bayly PV, Leigh MW, Knowles MR, Brody SL, Dutcher SK, Ferkol TW. Whole-exome capture and sequencing identifies HEATR2 mutation as a cause of primary ciliary dyskinesia. Am J Hum Genet. 2012;91(4):685-93. doi:10.1016/j.ajhg.2012.08.022  PMCID:PMC3484505  PMID:23040496  
  48. Rosenfeld M, Emerson J, McNamara S, Thompson V, Ramsey BW, Morgan W, Gibson RL, EPIC Study Group. Risk factors for age at initial Pseudomonas acquisition in the cystic fibrosis epic observational cohort. J Cyst Fibros. 2012;11(5):446-53. doi:10.1016/j.jcf.2012.04.003  PMID:22554417  
  49. Emond MJ, Louie T, Emerson J, Zhao W, Mathias RA, Knowles MR, Wright FA, Rieder MJ, Tabor HK, Nickerson DA, Barnes KC, National Heart, Lung, and Blood Institute (NHLBI) GO Exome Sequencing Project, Lung GO, Gibson RL, Bamshad MJ. Exome sequencing of extreme phenotypes identifies DCTN4 as a modifier of chronic Pseudomonas aeruginosa infection in cystic fibrosis. Nat Genet. 2012;44(8):886-9. doi:10.1038/ng.2344  PMCID:PMC3702264  PMID:22772370  
  50. Loeve M, Hop WC, de Bruijne M, van Hal PT, Robinson P, Aitken ML, Dodd JD, Tiddens HA, Computed Tomography Cystic Fibrosis Survival Study Group. Chest computed tomography scores are predictive of survival in patients with cystic fibrosis awaiting lung transplantation. Am J Respir Crit Care Med. 2012;185(10):1096-103. doi:10.1164/rccm.201111-2065OC  PMID:22403801  
  51. Knowles MR, Leigh MW, Carson JL, Davis SD, Dell SD, Ferkol TW, Olivier KN, Sagel SD, Rosenfeld M, Burns KA, Minnix SL, Armstrong MC, Lori A, Hazucha MJ, Loges NT, Olbrich H, Becker-Heck A, Schmidts M, Werner C, Omran H, Zariwala MA, Genetic Disorders of Mucociliary Clearance Consortium. Mutations of DNAH11 in patients with primary ciliary dyskinesia with normal ciliary ultrastructure. Thorax. 2012;67(5):433-41. doi:10.1136/thoraxjnl-2011-200301  PMID:22184204  
  52. Ferkol TW, Leigh MW. Ciliopathies: the central role of cilia in a spectrum of pediatric disorders. J Pediatr. 2012;160(3):366-71. doi:10.1016/j.jpeds.2011.11.024  PMCID:PMC3282141  PMID:22177992  
  53. Mayer-Hamblett N, Kronmal RA, Gibson RL, Rosenfeld M, Retsch-Bogart G, Treggiari MM, Burns JL, Khan U, Ramsey BW, EPIC Investigators. Initial Pseudomonas aeruginosa treatment failure is associated with exacerbations in cystic fibrosis. Pediatr Pulmonol. 2012;47(2):125-34. doi:10.1002/ppul.21525  PMCID:PMC3214247  PMID:21830317  
  54. Chuen-im P, Smyth MD, Segura B, Ferkol T, Rivera-Spoljaric K. Recurrent pleural effusion without intrathoracic migration of ventriculoperitoneal shunt catheter: a case report. Pediatr Pulmonol. 2012;47(1):91-5. doi:10.1002/ppul.21510  PMID:22170808  
  55. Schechter MS, McColley SA, Regelmann W, Millar SJ, Pasta DJ, Wagener JS, Konstan MW, Morgan WJ, Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Socioeconomic status and the likelihood of antibiotic treatment for signs and symptoms of pulmonary exacerbation in children with cystic fibrosis. J Pediatr. 2011;159(5):819-824.e1. doi:10.1016/j.jpeds.2011.05.005  PMID:21705017  
  56. Zariwala MA, Omran H, Ferkol TW. The emerging genetics of primary ciliary dyskinesia. Proc Am Thorac Soc. 2011;8(5):430-3. doi:10.1513/pats.201103-023SD  PMCID:PMC3209577  PMID:21926394  
  57. Farberman MM, Ibricevic A, Joseph TD, Akers KT, Garcia-Medina R, Crosby S, Clarke LL, Brody SL, Ferkol TW. Effect of polarized release of CXC-chemokines from wild-type and cystic fibrosis murine airway epithelial cells. Am J Respir Cell Mol Biol. 2011;45(2):221-8. doi:10.1165/rcmb.2009-0249OC  PMCID:PMC3266059  PMID:20639462  
  58. Treggiari, M. M., Retsch-Bogart, G., Mayer-Hamblett, N., Khan, U., Kulich, M., Kronmal, R., Williams, J., Hiatt, P., Gibson, R.L, Spencer, T., Orenstein, D., Chatfield, B. A., Froh, D. K., Burnes, J.L., Rosenfeld, M., Ramsey, B.W. forthe EPIC Investigators. Comparative efficacy and safety of four randomized regimens to treat early Pseudomonas aeruginosa infection in children with cystic fibrosis Archives of Pediatrics and Adolsescent Medicine. 2011;165(847):856. 
  59. Olin JT, Burns K, Carson JL, Metjian H, Atkinson JJ, Davis SD, Dell SD, Ferkol TW, Milla CE, Olivier KN, Rosenfeld M, Baker B, Leigh MW, Knowles MR, Sagel SD, Genetic Disorders of Mucociliary Clearance Consortium. Diagnostic yield of nasal scrape biopsies in primary ciliary dyskinesia: a multicenter experience. Pediatr Pulmonol. 2011;46(483):488. doi:10.1002/ppul.21402  PMID:21284095  
  60. Davis SD, Ferkol TW. Hitting the target: new treatments for cystic fibrosis. Am J Respir Crit Care Med. 2010;182(12):1460-1. doi:10.1164/rccm.201008-1311ED  PMID:21159902  
  61. Rosenfeld M, Emerson J, McNamara S, Joubran K, Retsch-Bogart G, Graff GR, Gutierrez HH, Kanga JF, Lahiri T, Noyes B, Ramsey B, Ren CL, Schechter M, Morgan W, Gibson RL, EPIC Study Group Participating Clinical Sites. Baseline characteristics and factors associated with nutritional and pulmonary status at enrollment in the cystic fibrosis EPIC observational cohort. Pediatr Pulmonol. 2010;45(9):934-44. doi:10.1002/ppul.21279  PMID:20597081  
  62. Flume PA, Mogayzel PJ Jr, Robinson KA, Rosenblatt RL, Quittell L, Marshall BC, Clinical Practice Guidelines for Pulmonary Therapies Committee, Cystic Fibrosis Foundation Pulmonary Therapies Committee. Cystic fibrosis pulmonary guidelines: pulmonary complications: hemoptysis and pneumothorax. Am J Respir Crit Care Med. 2010;182(3):298-306. doi:10.1164/rccm.201002-0157OC  PMID:20675678  
  63. Lie H, Zariwala MA, Helms C, Bowcock AM, Carson JL, Brown DE 3rd, Hazucha MJ, Forsen J, Molter D, Knowles MR, Leigh MW, Ferkol TW. Primary ciliary dyskinesia in Amish communities. J Pediatr. 2010;156(6):1023-5. doi:10.1016/j.jpeds.2010.01.054  PMCID:PMC2875274  PMID:20350728  
  64. Buff SM, Yu H, McCall JN, Caldwell SM, Ferkol TW, Flotte TR, Virella-Lowell IL. IL-10 delivery by AAV5 vector attenuates inflammation in mice with Pseudomonas pneumonia. Gene Ther. 2010;17(5):567-76. doi:10.1038/gt.2010.28  PMID:20357828  
  65. Farberman MM, Akers KT, Malone JP, Erdman-Gilmore P, Townsend RR, Ferkol T. Airway proteins involved in bacterial clearance susceptible to cathepsin G proteolysis. Eur Respir J. 2010;35(2):410-7. doi:10.1183/09031936.00020809  PMCID:PMC2818476  PMID:19679607  
  66. McDonald S, Wilson DB, Pumbo E, Kulkarni S, Mason PJ, Else T, Bessler M, Ferkol T, Shenoy S. Acquired monosomy 7 myelodysplastic syndrome in a child with clinical features suggestive of dyskeratosis congenita and IMAGe association. Pediatr Blood Cancer. 2010;54(1):154-7. doi:10.1002/pbc.22283  PMID:19760774  
  67. Gappa M, Ferkol T, Kovesi T, Landau L, McColley S, Sanchez I, Tal A, Wong GW, Zar H. Pediatric respiratory medicine--an international perspective. Pediatr Pulmonol. 2010;45(1):14-24. doi:10.1002/ppul.21165  PMID:20014351  
  68. Ferkol T, Zeitlin P, Abman S, Blaisdell CJ, O'Brodovich H. NHLBI training workshop report: The vanishing pediatric pulmonary investigator and recommendations for recovery. Pediatr Pulmonol. 2010;45(1):25-33. doi:10.1002/ppul.21155  PMID:20025052  
  69. Schechter MS, McColley SA, Silva S, Haselkorn T, Konstan MW, Wagener JS, Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis, North American Scientific Advisory Group for ESCF. Association of socioeconomic status with the use of chronic therapies and healthcare utilization in children with cystic fibrosis. J Pediatr. 2009;155(5):634-9.e1-4. doi:10.1016/j.jpeds.2009.04.059  PMID:19608199  
  70. Berk DR, Ciliberto HM, Sweet SC, Ferkol TW, Bayliss SJ. Aquagenic wrinkling of the palms in cystic fibrosis: comparison with controls and genotype-phenotype correlations. Arch Dermatol. 2009;145(11):1296-9. doi:10.1001/archdermatol.2009.260  PMID:19917960  
  71. Chen DL, Bedient TJ, Kozlowski J, Rosenbluth DB, Isakow W, Ferkol TW, Thomas B, Mintun MA, Schuster DP, Walter MJ. [18F]fluorodeoxyglucose positron emission tomography for lung antiinflammatory response evaluation. Am J Respir Crit Care Med. 2009;180(6):533-9. doi:10.1164/rccm.200904-0501OC  PMCID:PMC2742744  PMID:19574441  
  72. Bartlett JR, Friedman KJ, Ling SC, Pace RG, Bell SC, Bourke B, Castaldo G, Castellani C, Cipolli M, Colombo C, Colombo JL, Debray D, Fernandez A, Lacaille F, Macek M Jr, Rowland M, Salvatore F, Taylor CJ, Wainwright C, Wilschanski M, Zemková D, Hannah WB, Phillips MJ, Corey M, Zielenski J, Dorfman R, Wang Y, Zou F, Silverman LM, Drumm ML, Wright FA, Lange EM, Durie PR, Knowles MR, Gene Modifier Study Group. Genetic modifiers of liver disease in cystic fibrosis. JAMA. 2009;302(10):1076-83. doi:10.1001/jama.2009.1295  PMID:19738092  
  73. Leigh MW, Pittman JE, Carson JL, Ferkol TW, Dell SD, Davis SD, Knowles MR, Zariwala MA. Clinical and genetic aspects of primary ciliary dyskinesia/Kartagener syndrome. Genet Med. 2009;11(7):473-87. doi:10.1097/GIM.0b013e3181a53562  PMID:19606528  
  74. Treggiari MM, Rosenfeld M, Mayer-Hamblett N, Retsch-Bogart G, Gibson RL, Williams J, Emerson J, Kronmal RA, Ramsey BW, EPIC Study Group. Early anti-pseudomonal acquisition in young patients with cystic fibrosis: rationale and design of the EPIC clinical trial and observational study'. Contemp Clin Trials. 2009;30(3):256-68. doi:10.1016/j.cct.2009.01.003  PMCID:PMC2783320  PMID:19470318  
  75. Ferkol T, Zeitlin P, Abman S, Blaisdell CJ, O'Brodovich H. NHLBI training workshop report: the vanishing pediatric pulmonary investigator and recommendations for recovery. Lung. 2009;187(6):367-74. doi:10.1007/s00408-009-9184-0  PMID:19806399  
  76. Hillian, A.D., Londono, D., Dunn, J.M., Goddard, K.A., Pace, R.G., Knowles, M.R., Drumm, M.L., CF Gene Modifier Study Group. Modulation of cystic fibrosis lung disease by variants in interleukin-8 Genes and Immunity. 2008;9(501):508. 
  77. Elizur A, Cannon CL, Ferkol TW. Airway inflammation in cystic fibrosis. Chest. 2008;133(2):489-95. doi:10.1378/chest.07-1631  PMID:18252915  
  78. Moss RB, Milla C, Colombo J, Accurso F, Zeitlin PL, Clancy JP, Spencer LT, Pilewski J, Waltz DA, Dorkin HL, Ferkol T, Pian M, Ramsey B, Carter BJ, Martin DB, Heald AE. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Hum Gene Ther. 2007;18(8):726-32. doi:10.1089/hum.2007.022  PMID:17685853  
  79. Elizur A, Orscheln RC, Ferkol TW, Dunne WM Jr, Storch GA, Cannon CL. Transmission of Panton-Valentine leukocidin-positive Staphylococcus aureus between patients with cystic fibrosis. J Pediatr. 2007;151(1):90-2. doi:10.1016/j.jpeds.2007.04.016  PMID:17586197  
  80. Elizur A, Orscheln RC, Ferkol TW, Atkinson JJ, Dunne WM Jr, Buller RS, Armstrong JR, Mardis ER, Storch GA, Cannon CL. Panton-Valentine Leukocidin-positive methicillin-resistant Staphylococcus aureus lung infection in patients with cystic fibrosis. Chest. 2007;131(6):1718-25. doi:10.1378/chest.06-2756  PMID:17400676  
  81. Gregory AD, Hogue LA, Ferkol TW, Link DC. Regulation of systemic and local neutrophil responses by G-CSF during pulmonary Pseudomonas aeruginosa infection. Blood. 2007;109(8):3235-43. doi:10.1182/blood-2005-01-015081  PMCID:PMC1852251  PMID:17185469  
  82. Fennell PB, Quante J, Wilson K, Boyle M, Strunk R, Ferkol T. Use of high-dose ibuprofen in a pediatric cystic fibrosis center. J Cyst Fibros. 2007;6(2):153-8. doi:10.1016/j.jcf.2006.06.003  PMID:16844429  
  83. Sedor J, Hogue L, Akers K, Boslaugh S, Schreiber J, Ferkol T. Cathepsin-G interferes with clearance of Pseudomonas aeruginosa from mouse lungs. Pediatr Res. 2007;61(1):26-31. doi:10.1203/01.pdr.0000250043.90468.c2  PMID:17211136  
  84. Lie H, Ferkol T. Primary ciliary dyskinesia: recent advances in pathogenesis, diagnosis and treatment. Drugs. 2007;67(13):1883-92. PMID:17722956  
  85. Hardin DS, Adams-Huet B, Brown D, Chatfield B, Dyson M, Ferkol T, Howenstine M, Prestidge C, Royce F, Rice J, Seilheimer DK, Steelman J, Shepherds R. Growth hormone treatment improves growth and clinical status in prepubertal children with cystic fibrosis: results of a multicenter randomized controlled trial. J Clin Endocrinol Metab. 2006;91(12):4925-9. doi:10.1210/jc.2006-1101  PMID:17018651  
  86. Ferkol T, Leigh M. Primary ciliary dyskinesia and newborn respiratory distress. Semin Perinatol. 2006;30(6):335-40. doi:10.1053/j.semperi.2005.11.001  PMID:17142159  
  87. Bush A, Ferkol T. Movement: the emerging genetics of primary ciliary dyskinesia. Am J Respir Crit Care Med. 2006;174(2):109-10. doi:10.1164/rccm.2604002  PMID:16822932  
  88. Chen DL, Ferkol TW, Mintun MA, Pittman JE, Rosenbluth DB, Schuster DP. Quantifying pulmonary inflammation in cystic fibrosis with positron emission tomography. Am J Respir Crit Care Med. 2006;173(12):1363-9. doi:10.1164/rccm.200506-934OC  PMCID:PMC2662975  PMID:16543553  
  89. Ferkol T, Mitchison H, O'Callaghan C, Leigh M, Carson J, Lie H, Rosenbluth D, Brody SL. Current issues in the basic mechanisms, pathophysiology, diagnosis, and management of primary ciliary dyskinesia European Respiratory Monograph. 2006;37(291):313. 
  90. Ferkol T, Rosenfeld M, Milla CE. Cystic fibrosis pulmonary exacerbations. J Pediatr. 2006;148(2):259-64. doi:10.1016/j.jpeds.2005.10.019  PMID:16492439  
  91. Johnson MR, Ferkol TW, Shepherd RW. Energy cost of activity and exercise in children and adolescents with cystic fibrosis. J Cyst Fibros. 2006;5(1):53-8. doi:10.1016/j.jcf.2005.10.001  PMID:16360343  
  92. Virella-Lowell I, Zusman B, Foust K, Loiler S, Conlon T, Song S, Chesnut KA, Ferkol T, Flotte TR. Enhancing rAAV vector expression in the lung. J Gene Med. 2005;7(7):842-50. doi:10.1002/jgm.759  PMID:15838934  
  93. Hardin DS, Ferkol T, Ahn C, Dreimane D, Dyson M, Morse M, Prestidge C, Rice J, Seilheimer DK. A retrospective study of growth hormone use in adolescents with cystic fibrosis. Clin Endocrinol (Oxf). 2005;62(5):560-6. doi:10.1111/j.1365-2265.2005.02259.x  PMID:15853825  
  94. Joseph T, Look D, Ferkol T. NF-kappaB activation and sustained IL-8 gene expression in primary cultures of cystic fibrosis airway epithelial cells stimulated with Pseudomonas aeruginosa. Am J Physiol Lung Cell Mol Physiol. 2005;288(3):L471-9. doi:10.1152/ajplung.00066.2004  PMID:15516493  
  95. Hardin DS, Rice J, Ahn C, Ferkol T, Howenstine M, Spears S, Prestidge C, Seilheimer DK, Shepherd R. Growth hormone treatment enhances nutrition and growth in children with cystic fibrosis receiving enteral nutrition. J Pediatr. 2005;146(3):324-8. doi:10.1016/j.jpeds.2004.10.037  PMID:15756212  
  96. Drum, M.L., Konstan, M. W., Schluchter, M.D., Handler, A., Pace, R., Zou, F., Zarwala, M., Fargo, D., Goddard, K., Yankaskas, J.R., Wright, F.A., Knowles, MR. Genetic modifiers of lung disease in cystic fibrosis N Engl J Med. 2005;353(1443):1453. 
  97. Tosi MF, van Heeckeren A, Ferkol TW, Askew D, Harding CV, Kaplan JM. Effect of Pseudomonas-induced chronic lung inflammation on specific cytotoxic T-cell responses to adenoviral vectors in mice. Gene Ther. 2004;11(19):1427-33. doi:10.1038/sj.gt.3302290  PMID:15295614  
  98. Sirninger J, Muller C, Braag S, Tang Q, Yue H, Detrisac C, Ferkol T, Guggino WB, Flotte TR. Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector. Hum Gene Ther. 2004;15(9):832-41. doi:10.1089/hum.2004.15.832  PMID:15353038  
  99. Rosenbluth DB, Wilson K, Ferkol T, Schuster DP. Lung function decline in cystic fibrosis patients and timing for lung transplantation referral. Chest. 2004;126(2):412-9. doi:10.1378/chest.126.2.412  PMID:15302726  
  100. Van Heeckeren AM, Scaria A, Schluchter MD, Ferkol TW, Wadsworth S, Davis PB. Delivery of CFTR by adenoviral vector to cystic fibrosis mouse lung in a model of chronic Pseudomonas aeruginosa lung infection. Am J Physiol Lung Cell Mol Physiol. 2004;286(4):L717-26. doi:10.1152/ajplung.00227.2003  PMID:14514520  
  101. Richard JC, Chen DL, Ferkol T, Schuster DP. Molecular imaging for pediatric lung diseases. Pediatr Pulmonol. 2004;37(4):286-96. doi:10.1002/ppul.20024  PMID:15022124  
  102. Richard JC, Factor P, Ferkol T, Ponde DE, Zhou Z, Schuster DP. Repetitive imaging of reporter gene expression in the lung. Mol Imaging. 2003;2(4):342-9. PMID:14717333  
  103. Ferkol T, Cohn LA, Phillips TE, Smith A, Davis PB. Targeted delivery of antiprotease to the epithelial surface of human tracheal xenografts. Am J Respir Crit Care Med. 2003;167(10):1374-9. doi:10.1164/rccm.200209-1119OC  PMID:12615618  
  104. Richard JC, Zhou Z, Ponde DE, Dence CS, Factor P, Reynolds PN, Luker GD, Sharma V, Ferkol T, Piwnica-Worms D, Schuster DP. Imaging pulmonary gene expression with positron emission tomography. Am J Respir Crit Care Med. 2003;167(9):1257-63. doi:10.1164/rccm.200210-1217OC  PMID:12505860  
  105. Schuster DP, Kozlowski J, Hogue L, Ferkol TW. Imaging lung inflammation in a murine model of Pseudomonas infection: a positron emission tomography study. Exp Lung Res. 2003;29(45):57. 
  106. Aldallal N, McNaughton EE, Manzel LJ, Richards AM, Zabner J, Ferkol TW, Look DC. Inflammatory response in airway epithelial cells isolated from patients with cystic fibrosis. Am J Respir Crit Care Med. 2002;166(9):1248-56. doi:10.1164/rccm.200206-627OC  PMID:12403695  
  107. Ziady AG, Kelley TJ, Milliken E, Ferkol T, Davis PB. Functional evidence of CFTR gene transfer in nasal epithelium of cystic fibrosis mice in vivo following luminal application of DNA complexes targeted to the serpin-enzyme complex receptor. Mol Ther. 2002;5(4):413-9. doi:10.1006/mthe.2002.0556  PMID:11945068  
  108. Marshall LJ, Perks B, Ferkol T, Shute JK. IL-8 released constitutively by primary bronchial epithelial cells in culture forms an inactive complex with secretory component. J Immunol. 2001;167(5):2816-23. PMID:11509627  
  109. Ferkol TW, Look DC. Chinks in the armor of the airway: Pseudomonas infection in the cystic fibrosis lung. Am J Respir Cell Mol Biol. 2001;25(1):11-3. doi:10.1165/ajrcmb.25.1.f212  PMID:11472969  
  110. Mhanna MJ, Ferkol T, Martin RJ, Dreshaj IA, van Heeckeren AM, Kelley TJ, Haxhiu MA. Nitric oxide deficiency contributes to impairment of airway relaxation in cystic fibrosis mice. Am J Respir Cell Mol Biol. 2001;24(5):621-6. doi:10.1165/ajrcmb.24.5.4313  PMID:11350833  
  111. Gupta S, Eastman J, Silski C, Ferkol T, Davis PB. Single chain Fv: a ligand in receptor-mediated gene delivery. Gene Ther. 2001;8(8):586-92. doi:10.1038/sj.gt.3301451  PMID:11320404  
  112. Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M, Ferkol T, Flotte T, Muzyczka N. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol. 2000;74(18):8635-47. PMCID:PMC102071  PMID:10954565  
  113. Ferkol T, Eckman E, Swaidani S, Silski C, Davis P. Transport of bifunctional proteins across respiratory epithelial cells via the polymeric immunoglobulin receptor. Am J Respir Crit Care Med. 2000;161(3 Pt 1):944-51. doi:10.1164/ajrccm.161.3.9907018  PMID:10712347  
  114. van Heeckeren AM, Tscheikuna J, Walenga RW, Konstan MW, Davis PB, Erokwu B, Haxhiu MA, Ferkol TW. Effect of Pseudomonas infection on weight loss, lung mechanics, and cytokines in mice. Am J Respir Crit Care Med. 2000;161(1):271-9. doi:10.1164/ajrccm.161.1.9903019  PMID:10619831  
  115. Eckman EA, Mallender WD, Szegletes T, Silski CL, Schreiber JR, Davis PB, Ferkol TW. In vitro transport of active alpha(1)-antitrypsin to the apical surface of epithelia by targeting the polymeric immunoglobulin receptor. Am J Respir Cell Mol Biol. 1999;21(2):246-52. doi:10.1165/ajrcmb.21.2.3687  PMID:10423408  
  116. Chmiel JF, Drumm ML, Konstan MW, Ferkol TW, Kercsmar CM. Pitfall in the use of genotype analysis as the sole diagnostic criterion for cystic fibrosis. Pediatrics. 1999;103(4 Pt 1):823-6. PMID:10103316  
  117. Ziady AG, Ferkol T, Dawson DV, Perlmutter DH, Davis PB. Chain length of the polylysine in receptor-targeted gene transfer complexes affects duration of reporter gene expression both in vitro and in vivo. J Biol Chem. 1999;274(8):4908-16. PMID:9988733  
  118. Ziady AG, Ferkol T, Gerken T, Dawson DV, Perlmutter DH, Davis PB. Ligand substitution of receptor targeted DNA complexes affects gene transfer into hepatoma cells. Gene Ther. 1998;5(12):1685-97. doi:10.1038/sj.gt.3300777  PMID:10023448  
  119. Ferkol T, Mularo F, Hilliard J, Lodish S, Perales JC, Ziady A, Konstan M. Transfer of the human Alpha1-antitrypsin gene into pulmonary macrophages in vivo. Am J Respir Cell Mol Biol. 1998;18(5):591-601. doi:10.1165/ajrcmb.18.5.2874  PMID:9569229  
  120. van Heeckeren A, Ferkol T, Tosi M. Effects of bronchopulmonary inflammation induced by pseudomonas aeruginosa on adenovirus-mediated gene transfer to airway epithelial cells in mice. Gene Ther. 1998;5(3):345-51. doi:10.1038/sj.gt.3300593  PMID:9614554  
  121. Heeckeren A, Walenga R, Konstan MW, Bonfield T, Davis PB, Ferkol T. Excessive inflammatory response of cystic fibrosis mice to bronchopulmonary infection with Pseudomonas aeruginosa. J Clin Invest. 1997;100(11):2810-5. doi:10.1172/JCI119828  PMCID:PMC508486  PMID:9389746  
  122. Ziady AG, Perales JC, Ferkol T, Gerken T, Beegen H, Perlmutter DH, Davis PB. Gene transfer into hepatoma cell lines via the serpin enzyme complex receptor. Am J Physiol. 1997;273(2 Pt 1):G545-52. PMID:9277436  
  123. Perales JC, Grossmann GA, Molas M, Liu G, Ferkol T, Harpst J, Oda H, Hanson RW. Biochemical and functional characterization of DNA complexes capable of targeting genes to hepatocytes via the asialoglycoprotein receptor. J Biol Chem. 1997;272(11):7398-407. PMID:9054440  
  124. Flotte TR, Ferkol TW. Genetic therapy. Past, present, and future. Pediatr Clin North Am. 1997;44(1):153-78. PMID:9057789  
  125. Ferkol T, Pellicena-Palle A, Eckman E, Perales JC, Trzaska T, Tosi M, Redline R, Davis PB. Immunologic responses to gene transfer into mice via the polymeric immunoglobulin receptor. Gene Ther. 1996;3(8):669-78. PMID:8854092  
  126. Ferkol T, Perales JC, Mularo F, Hanson RW. Receptor-mediated gene transfer into macrophages. Proc Natl Acad Sci U S A. 1996;93(1):101-5. PMCID:PMC40186  PMID:8552583  
  127. Ferkol T, Perales JC, Eckman E, Kaetzel CS, Hanson RW, Davis PB. Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor. J Clin Invest. 1995;95(2):493-502. doi:10.1172/JCI117690  PMCID:PMC295497  PMID:7860731  
  128. Perales JC, Ferkol T, Molas M, Hanson RW. An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur J Biochem. 1994;226(2):255-66. PMID:8001543  
  129. Perales JC, Ferkol T, Beegen H, Ratnoff OD, Hanson RW. Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc Natl Acad Sci U S A. 1994;91(9):4086-90. PMCID:PMC43728  PMID:8171039  
  130. Margolis PA, Ferkol TW, Marsocci S, Super DM, Keyes LL, McNutt R, Harrell FE Jr. Accuracy of the clinical examination in detecting hypoxemia in infants with respiratory illness. J Pediatr. 1994;124(4):552-60. PMID:8151469  
  131. Ferkol T, McDowell KM, Gauderer MW, Alpert SE. Sinopulmonary manifestations of congenital bronchobiliary fistula. Clin Pediatr (Phila). 1994;33(3):181-4. PMID:8194299  
  132. Ferkol T, Kaetzel CS, Davis PB. Gene transfer into respiratory epithelial cells by targeting the polymeric immunoglobulin receptor. J Clin Invest. 1993;92(5):2394-400. doi:10.1172/JCI116845  PMCID:PMC288422  PMID:8227356  
  133. Ferkol T, Lindberg GL, Chen J, Perales JC, Crawford DR, Ratnoff OD, Hanson RW. Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASEB J. 1993;7(11):1081-91. PMID:8370479  
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