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Gene Therapy of Cystic Fibrosis Full Members

Name:  Mahmoud Abou Alaiwa, MD

Title:  Assistant Professor

Focus Area:  Dr. Alaiwa is a K08-funded Assistant Professor who also directs an Imaging Core in one of the Center’s CF-focused PPGs. His laboratory studies CFTR-dependent regulation of airway pH and how defects in CF affect innate immunity and the regulation of mucociliary transport.

Email:  Mahmoud-aboualaiwa@uiowa.edu

Office Number:  319-384-1107

Campus Address: 6338 PBDB

Richard C. Ahrens, MD Name: Richard C. Ahrens, MD

Title: Professor

Focus AreaDr. Ahrens has significant clinical research experience in the evaluation of pharmacologic treatments for CF, as well as in the clinical care of CF patients. His primary interests are: 1) the development of novel study designs to meet challenges in the pharmacologic management of CF pulmonary disease, and 2) in vivo and in vitro assessment of aerosol drug delivery to the lung.

Emailrichard-ahrens@uiowa.edu 

Office Number: (319) 356-4050

Campus Address: E-110-G GH
Name: Alejandro Comellas, MD

Title: Clinical Associate Professor

Focus Area: Dr. Comellas is Associate Director for Clinical Research Support in the UI Institute for Clinical and Translational Science. He has a broad background in clinical pulmonary medicine, specifically mechanisms of lung injury and repair, with an emphasis on distal lung progenitor cells and the development of strategies to improve antimicrobial activity in the airway.

EmailAlejandro-comellas@uiowa.edu

Office Number: 319-384-6484

Campus Address: W219-B GH
John F. Engelhardt, PhD NameJohn F. Engelhardt, PhD

Title: Professor

Focus Area:  Dr. Engelhardt’s research includes: 1) study of the molecular and cellular pathogenesis of CF lung disease and CF-related diabetes, 2) development of transgenic ferret animal models in which to study stem cell biology in the context of CF, including ferrets designed for lineage tracing of islet progenitors in the CF pancreas, and 3) development of gene therapies for CF lung and pancreatic disease. His laboratory is adept at the construction and use of many types of recombinant viral vectors (including adenovirus, AAV, lentivirus, retrovirus, and bocavirus). He also is PD/PI of a team science R24 that focuses on the pathogenesis of CF-related diabetes in CF ferrets and includes a clinical trial in CF infants and children <6 yrs of age. This grant has been converted into an NIDDK RC2 (application under review).

Emailjohn-engelhardt@uiowa.edu

Office Number: (319) 335-7744 

Campus Address: 1-111 BSB
Name: Anthony Fischer, MD, PhD

Title: Assistant Professor

Focus Area: Dr. Fischer is an Assistant Professor studying how mucins secreted by submucosal glands initiate the mucociliary transport of particles in the airway and how CF-associated alterations to mucin structure affect clearance and infection. His interests also include CF-associated adaptations of bacteria that allow for their persistence in the airway following CFTR modulator therapy.

Emailanthony-fischer @uiowa.edu

Office Number: 319-335-6992

Campus Address6312A PBDB

 
Name: Katherine Gibson-Corley, DVM, MS

Title: Associate  Professor

Focus Area: Dr. Gibson-Corley has nearly 15 years of experience in comparative pathology and translational research on animal models of human disease. As director of the P30 Comparative Pathology Core (Core-4), she will participate in many projects involving the CF pigs and ferrets. Her own research is focused on intestinal and pancreatic disease in these animal models.

Email:  katherine-gibson-corley@uiowa.edu

Office Number:  (319) 384-4430

Campus Address:  4184A MERF
Name: Eric A. Hoffman, PhD

Title: Professor

Focus Area: Dr. Hoffman’s research is driven by questions pertaining to lung physiology, and by the development of imaging software and equipment designed specifically for the study of these questions. He uses single and multi-spectral multidetector row spiral CT imaging methodology to objectively follow human lung pathology and pathophysiology, with a particular emphasis on lung diseases involving inflammation. His expertise has facilitated the evaluation of lung pathology and bacterial clearance in animal models of CF.

Email:  eric-hoffman@uiowa.edu 

Office Number:  (319) 356-1381 

Campus Address:  C748 GH  
Name: Douglas Hornick, MD

Title: Clinical Professor

Focus Area: Dr. Hornick is Director of the Adult CF Center and his participation in this P30 will facilitate the acquisition of specimens from CF patients and the recruitment of subjects for clinical studies. Dr. Hornick is also active in running clinical trials in CF patients, including testing of the efficacy of CFTR modulators and gallium nitrate in CF patients..

Email:  douglas-hornick@uiowa.edu

Office Number:  319-356-8266 

Campus Address: C-33C GH
Name: Yumi Imai, MD, PhD

Title: Associate  Professor

Focus Area: Dr. Imai aims to understand the mechanism underlying pancreatic b-cell failure in type 2 diabetes, including how lipid droplet proteins optimize intracellular lipid metabolism to support insulin secretion and mechanisms of islet inflammation. Her group discovered that the lipid droplet protein perilipin 5 plays an important role in supporting insulin secretion, and that it does so by regulating the metabolism of b-cell lipids. Her laboratory is defining the molecular targets of perilipin 5 act in augmenting insulin secretion, and her recruitment to the Center adds considerable strength to its CFRD research, given the links between pancreatic inflammation/fibrosis/adipogenesis and islet function in the CF ferret model.

Email:  yumi-imai@uiowa.edu

Office Number: 319-335-4844

Campus Address: 3318  PBDB
Name: Julia Klesney-Tait, MD

Title: Associate Professor

Focus Area: Dr. Klesney-Tait’s research focuses on the role of neutrophils in the regulation of inflammation and sepsis. In particular, she is interested in triggering receptor expressed on myeloid cells 1 (TREM-1), which is a component of the innate immune system, is expressed on neutrophils, and amplifies proinflammatory signaling and neutrophil migration.

Email:  julie-klesney-tait@uiowa.edu

Office Number: 319-356-3576

Campus Address: C34-12 GH

Name: Katie Larson-Ode, MD

Title:  Clinical Associate Professor

Focus Area: Dr. Larson-Ode was recruited to UI eight years ago to establish a CFRD presence and clinic. Since then she has been highly engaged in the ferret and pig CFRD research programs at UI. Her own research focuses on early CFRD pathogenesis in toddlers and young CF children, and she performed the first oral glucose tolerance study in this age bracket, confirming abnormalities that were first observed in CF ferrets. Dr. Larson-Ode was awarded the CF Foundation (CFF) “EnVision CF: Emerging Leaders in CF Endocrinology” career development grant and runs a CFF-funded multi-center clinical study on glucose tolerance in CF. Her clinical research and biobanked blood and plasma samples are key to the Center’s efforts in the identification of biomarkers of early islet failure in CF. These biomarkers are expected to be predictive of progression to CFRD in the longer term.

Email:  katie-larsonode@uiowa.edu

Office Number:  319-356-4443

Campus Address: 2022 BT

Name: David K. Meyerholz, DVM, PhD

Title: Professor

Focus Area: Dr. Meyerholz is Director of the Division of Comparative Pathology in the Department of Pathology. His research efforts are devoted mainly to the study of CF pig models involving gastrointestinal and reproductive pathologies. His leadership position in the Division of Comparative Pathology also has led to numerous collaborations in the area of CF pathophysiology.

Emaildavid-meyerholz@uiowa.edu 

Office Number: (319) 353-4589 

Campus Address: 1165 ML
Name: Paul B. McCray, Jr., MD

Title: Professor

Focus Area: Dr. McCray has a long-standing interest in CF, epithelial biology, genomics, innate immunity, and the application of gene transfer to CF lung disease. He is an expert in mucosal immunity and antimicrobial proteins involved in host-pathogen interactions. Dr. McCray also has a history of collaboration with Drs. Engelhardt, Sinn, Stoltz, Welsh, and Zabner. His research on lentiviral vectors and Piggybac transposon vectors for gene editing led to the development of new pseudotypes and strategies for lung- and liver-directed gene therapies. Dr. McCray directs a PPG focused on CF gene therapy.

Emailpaul-mccray@uiowa.edu

Office Number: (319) 335-6844

Campus Address: 6320 PBDB 
Name: Andrew Norris, MD, PhD

Title: Professor

Focus Area: Dr. Norris has broad expertise in the integrative physiology of diabetes. His laboratory studies the early pathogenesis of CFRD in ferret and pig models, with a focus on the extent to which redox stress, lipid abnormalities, and metabolic disease contribute to altered islet function in CFRD. Dr. Norris brings substantial expertise in the study of CFRD pathogenesis to the Center. He is PI on an R01 and an R24 (RC2 renewal pending) that are solely focused on CFRD research, and is co-I on the CFRD clinical trial at UI.

Emailandrew-norris@uiowa.edu

Office Number: (319) 335-7383

Campus Address: 1270B CBRB  
Name: Lynda S. Ostedgaard, PhD

Title: Research Associate Professor

Focus Area: Dr. Ostedgaard has a long-standing track record in the biology and biochemistry of CF disease, with a focus on the structure/function CFTR, the molecular basis of mucin abnormalities in the lung and intestine of the CF pig, and the creation of functional CFTR mini-genes for rAAV-directed gene therapy. 

Emaillynda-ostedgaard@uiowa.edu

Office Number: 319-335-7574

Campus Address: 6336 PBDB  

Name: Kalpaj Parekh, MD

Title:  Professor

Focus Area: Dr. Parekh’s laboratory focuses on the biology of endogenous airway epithelial stem cells in the development of obliterative bronchiolitis (OB) following lung transplantation. His R01-funded research investigates the mechanisms by which stem cell niches in the lung are destroyed in the transplanted lung (human and ferret), with the long-term goal of developing autologous stem cell-based therapies for the CF lung transplant. His research program has strong synergies with Dr. Engelhardt’s research on lung stem cells.

Email:  kalpaj-parekh@uiowa.edu

Office Number:  319-353-6758

Campus Address: SE521 GH

Name: Alejandro Pezzulo, MD

Title:  Assistant Professor

Focus Area: Dr. Pezzulo is physician scientists who recently acquired an NIH K01 to determine how HSP90 induces the hypersecretion of mucus and metaplasia of goblet cells in CF lung disease. He hypothesizes that inhibition of HSP90 induces transdifferentiation of goblet cells to ciliated cells by blocking ERBB2/EGFR signaling, and he is testing this possibility using lineage tracing. This pathway may also be important in the production of excessive and altered mucus by the CF intestine.

Email:  alejandro-pezzulo@uiowa.edu

Office Number:  319-335-2213

Campus Address: 6316 PBDB

Name: Philip Polgreen, MD, MPH

Title:  Professor

Focus Area: Dr. Polgreen’s research focuses on developing approaches to improve disease surveillance and to identify novel risk factors. His methodological approach, best described as computational epidemiology or data science, involves using both structured and unstructured data, building and using sensors, and running observational and small clinical trials. Although Dr. Polgreen’s work originally focused on infectious diseases, it has applications for chronic disease, and he has recently focused on understanding the extent to which risk for diabetes, pancreatitis, and kidney disease can be attributed to the CF-carrier state.

Email:  philip-polgreen@uiowa.edu

Office Number:  319-384-6194

Campus Address: SW34-Q GH

Name: Matthew Potthoff, PhD

Title:  Associate Professor

Focus Area: Dr. Potthoff’s research is focused on the physiologic mechanisms underlying hepatic glucose homeostasis and insulin sensitivity. He is particularly interested in the involvement of FGF21, which is produced by the acinar cells and is one of the first hormones whose levels decline during destruction of the exocrine pancreas in CF ferrets. Dr. Potthoff brings considerable expertise on mechanisms of pathogenesis in CFRD and pathways that alter glucose metabolism in CF ferret and pig models.

Email:  matthew-potthoff@uiowa.edu

Office Number:  319-384-4438

Campus Address: 3322 PBDB

Name: Kamal Rahmouni, PhD

Title:  Professor

Focus Area: Dr. Rahmouni’s research focuses on molecular and cellular regulation of altered glucose metabolism in diabetes. In collaboration with Drs. Norris and Engelhardt, he is studying how the central nervous system controls the secretion of pancreatic polypeptide (PP/PPY) by pancreatic islets and the implications of this effect for glucose metabolism in CFRD. His group is using CF and PPY-KO ferret models to study these processes, and hypothesize that altered innervation of the remodeled CF pancreas is responsible for reduced PP secretion in CF humans and ferrets.

Email:  kamal-rahmouni@uiowa.edu

Office Number:  319-353-5256

Campus Address: 2-248 BSB

Name: Christoph Randak, MD

Title: Associate Professor

Focus Area: Dr. Randak’s research, on the structural basis of CFTR gating and its dependence on the activity of adenylate kinase is shifting the current paradigm of how CFTR functions in vivo. His laboratory showed that the adenylate kinase inhibitor Ap5A potentiates activity of the F508del CFTR channel by interacting with CFTR residue Q1291. Using transgenic mice, he is evaluating the Q1291F CFTR mutant for the ability to rescue the intestinal phenotype of CFTR knockout mice and to reduce cholera toxin-induced loss of intestinal fluids. Dr. Randak was a recipient of the Harry Shwachman CF Clinical Investigator award and of an R01 grant. 

Email:  christoph-randak@uiowa.edu

Office Number:  319-335-6540

Campus Address: 6336 PBDB

Name: Joseph M. Reinhardt, MS, PhD

Title: Professor

Focus Area: Dr. Reinhardt uses imaging and image analysis to study normal lung function and to detect variations indicative of early disease. Using CT imaging and image registration, he has developed a set of textural and biomechanical features and machine learning to characterize lung disease involving CF, COPD, emphysema, and fibrosis. He collaborates with members of the Center in applying these technologies to the study of lung disease in the CF pig and ferret models.

Emailjoe-reinhardt@uiowa.edu

Office Number: (319) 335-5634

Campus Address: 5601 SC
Name: Kevin Rice, PhD

Title: Professor

Focus Area: Dr. Rice is a leader in the development of targeted delivery systems for gene transfer to the liver. His R01-funded research uses a high-affinity triantennary N-glycan attached to the PEG-peptide to trigger hepatocytic endocytosis of nanoparticles via the asialoglycoprotein receptor. His group also focuses on developing nanoparticles that target somatostatin receptor-expressing cells in the periphery. As this receptor is expressed on exocrine acinar cells of the pancreas, it represents unique opportunities for gene therapy of CFRD in the CF ferret and pig models.

Email: kevin-rice@uiowa.edu

Office Number:
(319) 335-9903 

Campus Address: 300 PHAR  

Name: Thomas Rutkowski, PhD

Title:  Associate Professor

Focus Area: Dr. Rutkowski seeks to understand how the endoplasmic reticulum (ER) regulates lipid metabolism in hepatocytes in the setting of chronic stress. His group demonstrated that perturbations of the ER disrupt lipid metabolism by the liver, and that this leads to fat accumulation (steatosis). His group is determining how activation of the unfolded protein response (UPR) during fasting/feeding cycles contributes to normal homeostatic regulation of lipid metabolism, and how the inhibition of fatty acid beta-oxidation by the UPR protects ER functions. Hepatic steatosis is a complication in CF patients and ferrets, and may contribute to altered glucose metabolism in CFRD.

Email:  thomas-rutkowski@uiowa.edu

Office Number:  319-335-7739

Campus Address: 1-570 BSB

Name: Julien Sebag, PhD

Title: Assistant Professor

Focus Area: Dr. Sebag’s laboratory studies how MRAP2 regulates the actions of ghrelin, a peptide hormone that can inhibit insulin secretion by binding to the ghrelin receptor in d-cells of the pancreatic islet. By deleting MRAP2 in the d-cells of mice, this group showed that MRAP2 is required for ghrelin-mediated inhibition of insulin secretion. His group is developing small-molecule enhancers of insulin-stimulated GLUT4 translocation for the treatment of insulin resistance in diabetes. Studies by Dr. Norris have linked changes in acylated-ghrelin in the CF ferret model to impaired insulin secretion and glucose metabolism. 

Email:  julien-sebag@uiowa.edu

Office Number:  319-335-3270

Campus Address:  3330 PBDB

Name: Patrick L. Sinn, PhD

Title: Associate Professor

Focus Area: Dr. Sinn's research focuses on developing integrating vector systems for the treatment of CF lung disease. His R01-funded research focuses on the development of a piggyBac hybrid vector system that integrates into airway epithelial cells, and the engineering of transposases and zinc-fingers to integrate transgenes into safe-harbor genomic loci. These systems, in addition to novel lentiviral pseudotypes developed by Dr. Sinn, are being used for lung-directed gene therapy in the CF pig and ferret models.

Emailpatrick-sinn@uiowa.edu

Office Number: (319) 335-8190 

Campus Address: 6318 PBDB  
Name: David A. Stoltz, MD, PhD

Title:  Professor

Focus Area: Dr. Stoltz is Director of the CFF RDP and an NIH PPG studying the pathogenesis of CF lung disease in CF pig models. His areas of research include the roles of CFTR in smooth muscle, and CF disease pathogenesis involving goblet cell functions, innate immunity, and inflammation. Recently, Dr. Stoltz developed in vitro organoid models of the gallbladder from CF pigs, ferrets, and humans, which his group uses to study how CFTR function influences pH regulation and mucin secretion in this organ.

Emaildavid-stoltz@uiowa.edu

Office Number:  319-384-1107

Campus Address: 6322 PBDB

Name: Eric Taylor, PhD

Title:  Associate Professor

Focus Area: Dr. Taylor’s laboratory studies the molecular mechanisms that regulate mitochondrial function in hepatocytes and gluconeogenesis in the liver. Specifically, he studies the relationship between the function of the mitochondrial pyruvate carrier (MPC) protein in the liver and pathologically elevated gluconeogenesis in diabetes. His laboratory has an additional interest in problems related to skeletal muscle function in diabetes. Dr. Taylor brings new strengths in the area of hepatic glucose metabolism to the Center’s CFRD research.

Email:  eric-taylor@uiowa.edu

Office Number:  319-384-4098

Campus Address: 3316 PBDB

Name: Peter Thorne, PhD

Title: Professor

Focus Area: Dr. Thorne's research expertise includes the development of aerosal-based techniques for delivering therapeutic agents and gene therapy vectors to the lung, as well as studies of innate immune responses to inhaled biological materials. His group collaborates with Drs. Stoltz, Welsh, and Engelhardt in both the CF ferret and pig models. Additionally, he is co-I on a recently acquired gene-editing grant in CF pigs awarded to Dr. McCray.

Emailpeter-thorne@uiowa.edu

Office Number: (319) 335-4216

Campus Address: S341A CPHB

Name: Aliye Uc, MD

Title:  Professor

Focus Area: Dr. Uc’s research focuses on CF and non-CF exocrine pancreatic disease, CFRD, and rAAV-mediated gene therapy to the pancreas, using CF pig and ferret models. Her clinical research includes the study of acute and chronic pancreatitis in children, and she directs a U01 consortium on pediatric chronic pancreatitis. This U01 provides considerable translational strengths pertaining to modifier genes that affect CF pancreatic disease. In this context, Dr. Uc has generated a knockout ferret (SPINK1 gene) that models inherited chronic pancreatitis, in collaboration with the Center’s Animal Model Core, and her group is using this model to understand overlapping CFTR-independent mechanisms of pancreatitis-associated diabetes. She collaborates heavily with Drs. Engelhardt and Norris on dissecting the pathogenesis of CFRD within R01- and R24-funded programs.

Email:  aliye-uc@uiowa.edu

Office Number:  319-384-6032

Campus Address: 1120-C BT

Name: Michael J. Welsh, MD

Title: Professor

Focus Area: Dr. Welsh has a long-standing interest in the study and treatment of CF. His laboratory uses a multi-faceted approach to understand the biology of CF, with several areas of emphasis. The first is an investigation of the structure and function of CFTR. The second is an exploration of how CFTR defects lead to impairment of innate immunity in the airway and to lung disease. To facilitate this research, his group generated a number of porcine CF models. These have brought a new understanding of the disease and are aiding in the development of novel therapies in collaboration with Drs. Stoltz, Zabner, McCray, Abou Alai, Fischer, and many other Center Members.

Emailmichael-welsh@uiowa.edu 

Office Number: (319) 335-7619 

Campus Address: 6332 PBDB  
Name: Timothy L. Yahr, PhD

Title: Professor

Focus Area: Dr. Yahr’s research program focuses primarily on the type III secretion system (T3SS) of Pseudomonas aeruginosa, with the general aims of defining regulatory mechanisms that control expression of T3SS genes as well as the roles of the T3SS in pathogenesis. He has broad training in microbial genetics and physiology, protein biochemistry, protein transport systems, and microbial pathogenesis.

Emailtim-yahr@uiowa.edu

Office Number: (319) 335-9688

Campus Address: 200A EMRB
Name: Ziying Yan, PhD 

Title: Research Associate Professor

Focus Area: Dr. Yan's research pertains to the molecular biology of rAAV and human Bocaviruses (HBoV), and to their development as recombinant viral vectors for use in gene therapy of CF. Most recently, he generated new recombinant HBoV serotypes with an intestinal tropism. Through his CF Foundation-funded research, he is generating a novel genetic ferret model with targeted integration of a CFTR mini-exon. This model will make it possible to test gene-editing strategies in vivo. Dr. Yan also collaborates with Drs. Engelhardt, McCray, and Sinn in testing vectors in the CF ferret models.

Emailziying-yan@uiowa.edu

Office Number: (319) 335-9855

Campus Address: 1-111 BSB  
Name: Joseph Zabner, MD

Title: Professor

Focus Area: Dr. Zabner has significant experience in basic CF research, clinical CF research, and the care of patients with CF. His interests include gene therapy for CF lung disease and mechanisms by which CFTR regulates airway fluid composition and innate immunity, and his research elegantly bridges studies in CF patients with those in CF pig animal models.

Emailjoseph-zabner@uiowa.edu

Office Number: 319-356-4419 

Campus Address: 6324 PBDB