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Principal Investigator: Raouf S. Amin
Organization: CINCINNATI CHILDRENS HOSP MED CTR
Fiscal Year: 2024
Award: $944,438
Funding agency: National Institute of Diabetes and Digestive and Kidney Diseases
PROJECT SUMMARY – OVERVIEW
Cystic Fibrosis results from the absence or dysfunction of the Cystic Fibrosis Transmembrane Conductance
Regulator (CFTR) chloride channel. Defects in CFTR are strongly associated with genetic variations in the CFTR
gene that are unique to individual CF patients or to subgroups within the CF patient population. Thus, a
personalized medicine approach helps customize care to individual CF patients based on the unique molecular
and phenotypic characteristics associated with their CFTR gene. In this renewal application we will promote
clinical, translational, and basic research collaboration to improve CF therapy and patient quality of life through
personalized medicine with an emphasis to address CF minority population. This goal will be achieved through
3 integrated cores: Personalized Clinical Core, Personalized Model Systems Core and the new CF-Organ-on-a
Chip Core. This will drive successful therapeutic outcomes and minimize potential adverse effects of chronic
therapies. Given that the largest benefit of highly effective modulators is on lung function with little tangible effect
on CF related diabetes, the focus of the CF-Organ-on-a Chip Core will be the study of mechanisms of diabetes
in ductal and endocrine pancreatic cells lacking CFTR in multiple genetic mutations. We will leverage an IRB
approved genotype-phenotype database available for clinical and research use, to develop Personalized Cystic
Fibrosis Therapy and Research Center (PCFC) for individual patients to (i) determine his/her susceptibility to a
particular form of CF mutation, and ii) take steps to mitigate the early onset of the disease. Finally, the future of
CF research offers the potential for a variety of CFTR-targeted therapies (i e., CFTR modulators) to treat the
underlying cause of CF, with multiple modulators currently in different stages of development.
Terms: <21+ years old><Address><Adult><Adult Human><Adverse effects><Applications Grants><Assay><Autograft><Autologous Transplantation><Autotransplant><B9 endocrine pancreas><Basic Research><Basic Science><Bioassay><Biologic Models><Biological><Biological Assay><Biological Models><Bronchoscopy><CF patients><CF related diabetes><CFTR><CFTR Protein><COVID-19><CV-19><California><Caring><Caucasian><Caucasian Race><Caucasians><Caucasoid><Caucasoid Race><Cell Body><Cell secretion><Cells><Cellular Secretion><Center Core Grants><Characteristics><Childhood><Children's Hospital><Chloride Channels><Chloride Ion Channels><Chlorides><Chronic><Clinical><Clinical Research><Clinical Sciences><Clinical Study><Collaborations><Conduct Clinical Trials><Core Facility><Coronavirus Infectious Disease 2019><Cystic Fibrosis><Cystic Fibrosis Transmembrane Conductance Regulator><DNA Alteration><DNA Sequence Alteration><DNA mutation><Data Bases><Databases><Dedications><Defect><Development><Diabetes Mellitus><Doctor of Philosophy><Duct><Duct (organ) structure><Duodenum><Dysfunction><EMR system><Emotional well being><Endocrine><Endocrine Pancreas><Endoscopy><Enteral><Enteric><Epithelial Cells><Evaluation><Faculty><Feels well><Fluids and Secretions><Fostering><Functional disorder><Funding><Future><Gases><Genetic Alteration><Genetic Change><Genetic Diversity><Genetic Variation><Genetic defect><Genetic mutation><Genotype><Goals><Grant><Grant Proposals><Healthcare><Hispanic><Home><Hospitals><Human><IRB><IRBs><Individual><Individuals from minority><Individuals of minority><Institution><Institutional Review Boards><Intestinal><Intestines><Investigators><Islands of Langerhans><Islet Cell><Islets of Langerhans><Laboratories><Liver><Lung><Lung Grafting><Lung Respiratory System><Lung Surfactant><Lung Transplantation><MR Imaging><MR Tomography><MRI><MRIs><Magnetic Resonance Imaging><Measurement><Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance><Medical center><Medicine><Minority Groups><Minority People><Minority Population><Minority individual><Model System><Modern Man><Molecular><Mucoviscidosis><Mutation><NIDDK><NMR Imaging><NMR Tomography><Nasal><Nasal Epithelium><Nasal Passages Nose><National Institute of Diabetes and Digestive and Kidney Diseases><Nesidioblasts><Non-Hispanic><Nonhispanic><Normal mental condition><Normal mental state><Normal psyche><Nose><Not Hispanic or Latino><Nuclear Magnetic Resonance Imaging><Occidental><Onset of illness><Oral Polio Vaccine><Oral Poliovirus Vaccine><Organoids><Outcome><P30 Award><P30 Grant><P30 Mechanism><P30 Program><Pancreas><Pancreatic><Pancreatic Islets><Pars endocrina pancreatis><Patient Monitoring><Patients><Pediatric Hospitals><Pediatrics><Ph.D.><PhD><Phenotype><Physicians><Physiopathology><Pneumology><Pneumonology><Population><Position><Positioning Attribute><Predisposition><Productivity><Progenitor Cells><Psychological Well Being><Pulmonary Graft><Pulmonary Medicine><Pulmonary Surfactants><Pulmonary Transplant><Pulmonary Transplantation><Pulmonology><QOC><QOL><Quality of Care><Quality of life><Rectum><Regulator Genes><Reporting><Research><Research Activity><Research Personnel><Research Resources><Researchers><Resources><Respiratory System, Nose, Nasal Passages><Rotavirus Vaccines><Sabin Vaccine><Sampling><Scientist><Sense of well-being><Sequence Alteration><Services><Site><Structure><Subgroup><Susceptibility><Technology><Time><Total Pancreatectomy><Training><Transcriptional Regulatory Elements><Translational Research><Translational Science><Underrepresented Ethnic Minority><Underrepresented Minority><United States><Well in self><Xe element><Xenon><Zeugmatography><accurate diagnosis><adulthood><autologous graft><autotransplantation><biobank><biologic><biorepository><bowel><bronchial epithelium><cohort><coronavirus disease 2019><coronavirus disease-19><coronavirus infectious disease-19><cystic fibrosis patients><cystic fibrosis related diabetes><cystic fibrosis transmembrane regulator><data base><database of Genotypes and Phenotypes><dbGaP><demographics><developmental><diabetes><disease onset><disorder onset><disparity in health><early onset><electronic medical record system><electronic medical system><emotional wellbeing><emotional wellness><endoscopic imaging><gastrointestinal><genome mutation><genomic alteration><health care><health care service><health disparity><healthcare service><hepatic body system><hepatic organ system><homes><imaging study><improved><individual patient><individuals with CF><individuals with cystic fibrosis><interest><islet progenitor><lung function><lung health><lung transplant><mental well-being><mental wellbeing><mental wellness><multidisciplinary><news><organ on a chip><organ on chip><pancreas total excision><pathophysiology><patient population><patients with CF><patients with cystic fibrosis><pediatric><personalization of treatment><personalized medicine><personalized therapy><personalized treatment><preservation><programs><psychological wellbeing><psychological wellness><pulmonary><pulmonary function><pulmonary health><recruit><regulatory gene><research facility><self wellness><sense of wellbeing><stem cell technology><stem cells><success><synergism><targeted drug therapy><targeted drug treatments><targeted therapeutic><targeted therapeutic agents><targeted therapy><targeted treatment><therapeutic outcome><therapy outcome><trans acting element><translation research><translational investigation><under-representation of minorities><under-represented minority><underrepresentation of minorities><white race>