Personalized Cystic Fibrosis Therapy and Research Center
个性化囊性纤维化治疗和研究中心
基本信息
- 批准号:10672703
- 负责人:
- 金额:$ 96.56万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2018
- 资助国家:美国
- 起止时间:2018-08-01 至 2025-06-30
- 项目状态:未结题
- 来源:
- 关键词:AddressAdultAdverse effectsApplications GrantsAutologous TransplantationBasic ScienceBiologicalBiological AssayBiological ModelsBronchoscopyCaliforniaCaringCaucasiansCell secretionCellsCenter Core GrantsCharacteristicsChildhoodChloride ChannelsChloridesChronicClinicalClinical ResearchClinical SciencesCollaborationsConduct Clinical TrialsCore FacilityCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDNA Sequence AlterationDatabasesDedicationsDefectDevelopmentDiabetes MellitusDoctor of PhilosophyDuct (organ) structureDuodenumEndocrineEndoscopyEnteralEpithelial CellsEvaluationFacultyFluids and SecretionsFosteringFunctional disorderFundingFutureGasesGenetic VariationGenotypeGoalsGrantHealthcareHispanicHomeHospitalsHumanIndividualInstitutionInstitutional Review BoardsIntestinesIslet CellIslets of LangerhansLaboratoriesLiverLungLung TransplantationMagnetic Resonance ImagingMeasurementMedical centerMedicineMinority GroupsMolecularMutationNasal EpitheliumNational Institute of Diabetes and Digestive and Kidney DiseasesNoseNot Hispanic or LatinoOnset of illnessOral Poliovirus VaccineOrganoidsOutcomePancreasPatient MonitoringPatientsPediatric HospitalsPediatricsPhenotypePhysiciansPopulationPositioning AttributePredispositionProductivityPulmonary SurfactantsPulmonologyQuality of CareQuality of lifeRectumRegulator GenesReportingResearchResearch ActivityResearch PersonnelResourcesRotavirus VaccinesSamplingScientistServicesSiteStructureSubgroupTechnologyTimeTotal PancreatectomyTrainingTranslational ResearchUnderrepresented MinorityUnited StatesWell in selfXenonaccurate diagnosisbiobankbronchial epitheliumcohortcoronavirus diseasecystic fibrosis patientscystic fibrosis related diabetesdatabase of Genotypes and Phenotypesdemographicsearly onsetelectronic medical record systemgastrointestinalhealth care servicehealth disparityimaging studyimprovedindividual patientinterestlung healthmultidisciplinarynewsorgan on a chippatient populationpersonalized medicinepreservationprogramspulmonary functionrecruitrectalresearch facilitystem cell technologystem cellssuccesssynergismtargeted treatmenttherapy outcome
项目摘要
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.
项目概要-概述
囊性纤维化是由囊性纤维化跨膜传导的缺失或功能障碍引起的。
调节器(CFTR)氯离子通道。CFTR的缺陷与CFTR的遗传变异密切相关
基因是个体CF患者或CF患者群体内的亚组所特有的。因此
个性化的医疗方法有助于根据独特的分子生物学特性为个体CF患者定制护理。
和与其CFTR基因相关的表型特征。在此更新应用程序中,我们将促进
临床,转化和基础研究合作,以改善CF治疗和患者的生活质量,
个性化医疗,重点是解决CF少数群体。这一目标将通过
3个集成核心:个性化临床核心,个性化模型系统核心和新的CF-器官上
芯片核心。这将推动成功的治疗结果,并最大限度地减少慢性炎症的潜在不良影响。
治疗鉴于高效调节剂的最大益处是对肺功能的影响,
在CF相关糖尿病方面,CF-Organ-on-a Chip Core的重点将是研究糖尿病的机制
在多个基因突变中缺乏CFTR的导管和内分泌胰腺细胞中。我们将利用内部审查委员会
获批的基因型-表型数据库可用于临床和研究,以开发个性化的囊性
纤维化治疗和研究中心(PCFC)为个体患者(i)确定他/她对一种或多种抗纤维化药物的易感性,
特定形式的CF突变,和ii)采取措施减轻疾病的早期发作。最后,未来
CF研究为各种CFTR靶向治疗提供了潜力(即,CFTR调节剂)来治疗
CF的根本原因,目前有多种调节剂处于不同的开发阶段。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
数据更新时间:{{ journalArticles.updateTime }}
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
数据更新时间:{{ journalArticles.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ monograph.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ sciAawards.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ conferencePapers.updateTime }}
{{ item.title }}
- 作者:
{{ item.author }}
数据更新时间:{{ patent.updateTime }}
Raouf S. Amin其他文献
Upper Airway Volume Segmentation Analysis Using Cine MRI Findings in Children with Tracheostomy Tubes
使用气管切开插管儿童的电影 MRI 结果进行上气道体积分割分析
- DOI:
- 发表时间:
2007 - 期刊:
- 影响因子:4.8
- 作者:
Bradley L. Fricke;M. Bret Abbott;Lane F. Donnelly;B. Dardzinski;S. Poe;M. Kalra;Raouf S. Amin;Robin T. Cotton - 通讯作者:
Robin T. Cotton
Chapter 14 – Chronic Respiratory Failure
第 14 章 – 慢性呼吸衰竭
- DOI:
- 发表时间:
2006 - 期刊:
- 影响因子:0
- 作者:
Raouf S. Amin - 通讯作者:
Raouf S. Amin
Obliterative bronchiolitis in a 13-year-old pre-transplant cystic fibrosis patient
- DOI:
10.1016/j.jcf.2007.05.006 - 发表时间:
2008-01-01 - 期刊:
- 影响因子:
- 作者:
Patrick O. Sobande;James D. Acton;Raouf S. Amin;Jeanne Weiland - 通讯作者:
Jeanne Weiland
Raouf S. Amin的其他文献
{{
item.title }}
{{ item.translation_title }}
- DOI:
{{ item.doi }} - 发表时间:
{{ item.publish_year }} - 期刊:
- 影响因子:{{ item.factor }}
- 作者:
{{ item.authors }} - 通讯作者:
{{ item.author }}
{{ truncateString('Raouf S. Amin', 18)}}的其他基金
Randomized Control Trial of oxygen therapy in Children and Adolescents with Down Syndrome and Obstructive Sleep Apnea
唐氏综合症和阻塞性睡眠呼吸暂停儿童和青少年氧疗的随机对照试验
- 批准号:
10838939 - 财政年份:2022
- 资助金额:
$ 96.56万 - 项目类别:
Randomized Control Trial of oxygen therapy in Children and Adolescents with Down Syndrome and Obstructive Sleep Apnea
唐氏综合症和阻塞性睡眠呼吸暂停儿童和青少年氧疗的随机对照试验
- 批准号:
10518497 - 财政年份:2022
- 资助金额:
$ 96.56万 - 项目类别:
Personalized Cystic Fibrosis Therapy and Research Center
个性化囊性纤维化治疗和研究中心
- 批准号:
10672708 - 财政年份:2018
- 资助金额:
$ 96.56万 - 项目类别:
Regional monitoring of CF lung disease after changes in mechanical airway-clearance treatment
机械气道清除治疗改变后 CF 肺部疾病的区域监测
- 批准号:
10737221 - 财政年份:2016
- 资助金额:
$ 96.56万 - 项目类别:
UTE MRI to monitor CF lung disease and response to CFTR modulation in young children
UTE MRI 用于监测幼儿 CF 肺部疾病和对 CFTR 调节的反应
- 批准号:
9896865 - 财政年份:2016
- 资助金额:
$ 96.56万 - 项目类别:
Passive stretch of the chest wall in patients with Congential Muscular Dystrophy
先天性肌营养不良症患者的胸壁被动拉伸
- 批准号:
8445541 - 财政年份:2013
- 资助金额:
$ 96.56万 - 项目类别:
Passive stretch of the chest wall in patients with Congential Muscular Dystrophy
先天性肌营养不良症患者的胸壁被动拉伸
- 批准号:
8708194 - 财政年份:2013
- 资助金额:
$ 96.56万 - 项目类别:
Cincinnati Children's Summer Medical Student Respiratory Research Fellowship
辛辛那提儿童夏季医学生呼吸研究奖学金
- 批准号:
10397502 - 财政年份:2012
- 资助金额:
$ 96.56万 - 项目类别:
Cincinnati Children's Summer Medical Student Respiratory Research Fellowship
辛辛那提儿童夏季医学生呼吸研究奖学金
- 批准号:
10630069 - 财政年份:2012
- 资助金额:
$ 96.56万 - 项目类别:
Dynamic Computational Modeling of Obstructive Sleep Apnea in Down Syndrome
唐氏综合症阻塞性睡眠呼吸暂停的动态计算模型
- 批准号:
8323924 - 财政年份:2010
- 资助金额:
$ 96.56万 - 项目类别:
相似海外基金
Co-designing a lifestyle, stop-vaping intervention for ex-smoking, adult vapers (CLOVER study)
为戒烟的成年电子烟使用者共同设计生活方式、戒烟干预措施(CLOVER 研究)
- 批准号:
MR/Z503605/1 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Research Grant
Early Life Antecedents Predicting Adult Daily Affective Reactivity to Stress
早期生活经历预测成人对压力的日常情感反应
- 批准号:
2336167 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Standard Grant
RAPID: Affective Mechanisms of Adjustment in Diverse Emerging Adult Student Communities Before, During, and Beyond the COVID-19 Pandemic
RAPID:COVID-19 大流行之前、期间和之后不同新兴成人学生社区的情感调整机制
- 批准号:
2402691 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Standard Grant
Elucidation of Adult Newt Cells Regulating the ZRS enhancer during Limb Regeneration
阐明成体蝾螈细胞在肢体再生过程中调节 ZRS 增强子
- 批准号:
24K12150 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Migrant Youth and the Sociolegal Construction of Child and Adult Categories
流动青年与儿童和成人类别的社会法律建构
- 批准号:
2341428 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Standard Grant
Understanding how platelets mediate new neuron formation in the adult brain
了解血小板如何介导成人大脑中新神经元的形成
- 批准号:
DE240100561 - 财政年份:2024
- 资助金额:
$ 96.56万 - 项目类别:
Discovery Early Career Researcher Award
Laboratory testing and development of a new adult ankle splint
新型成人踝关节夹板的实验室测试和开发
- 批准号:
10065645 - 财政年份:2023
- 资助金额:
$ 96.56万 - 项目类别:
Collaborative R&D
Usefulness of a question prompt sheet for onco-fertility in adolescent and young adult patients under 25 years old.
问题提示表对于 25 岁以下青少年和年轻成年患者的肿瘤生育力的有用性。
- 批准号:
23K09542 - 财政年份:2023
- 资助金额:
$ 96.56万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Identification of new specific molecules associated with right ventricular dysfunction in adult patients with congenital heart disease
鉴定与成年先天性心脏病患者右心室功能障碍相关的新特异性分子
- 批准号:
23K07552 - 财政年份:2023
- 资助金额:
$ 96.56万 - 项目类别:
Grant-in-Aid for Scientific Research (C)
Issue identifications and model developments in transitional care for patients with adult congenital heart disease.
成人先天性心脏病患者过渡护理的问题识别和模型开发。
- 批准号:
23K07559 - 财政年份:2023
- 资助金额:
$ 96.56万 - 项目类别:
Grant-in-Aid for Scientific Research (C)