Initiation of an in Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via Induced Pluripotent Stem Cell Technology
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Obtention of induced pluripotent stem cell line (iPS): yes/no
研究概览
简要总结
In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
详细描述
The investigator's primary objective is to generate iPS lines from 3 CF patients and from 3 healthy subjects.
Secondary objectives include verification that cell lines express the CFTR gene according to their genotype, verification or relative production of the CFTR protein for each iPS line, and amplification of obtain iPS lines for aliquot creation to facilitate sharing.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- •Pregnancy, breastfeeding
- •Participant in an exclusion period determined by a previous study
- •Participant under any kind of guardianship
- •Unaffiliated with or not a beneficiary of a social security program (health insurance)
- •Subject deprived of liberty (e.g. prisoners)
- •Subject with positive infectious markers for HIV1, HIV2, HBC or HBV
- •Exclusion Criteria for subjects with Cystic Fibrosis:
- •Any pathology requiring a treatment or a pathology not requiring treatment but with clinical significance according to the investigator
结局指标
主要结局
Obtention of induced pluripotent stem cell line (iPS): yes/no
时间窗: 28 days
Was a pluripotent stem cell line obtained? yes/no
次要结局
- Functional bronchial epithelium present for the iPS? yes/no(28 days)
- Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no(28 days)