In Vitro Model of the Cystic Fibrosis Bronchial Epithelium Via iPS Technology

University Hospital, Montpellier6 enrolled

Overview

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.

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Cystic Fibrosis

Eligibility

Sex: ALLMin age: 18 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria for subjects with Cystic Fibrosis: * Homozygote for the p.Phe508del mutation * Signed informed consent given by the subject Inclusion Criteria for subjects without Cystic Fibrosis: * Signed informed consent given by the subject Exclusion Criteria: * 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

Locations (1)

Hôpital Arnaud de Villeneuve - CHU de Montpellier

Montpellier, France

Outcomes

Primary Outcomes

Obtention of induced pluripotent stem cell line (iPS): yes/no

Was a pluripotent stem cell line obtained? yes/no

Time frame: 28 days

Secondary Outcomes

Functional bronchial epithelium present for the iPS? yes/no

Was a functional bronchial epithelium present for the iPS? yes/no

Time frame: 28 days

Cystic fibrosis transmembrane conductance regulator (CFTR) channel function: yes/no

CFTR channel function demonstrated via response following exposure to modified oligonucleotides targeting the mutated CFTR transcript.

Time frame: 28 days

Data from ClinicalTrials.gov

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