Pathology of Helicases and Premature Aging: Study by Derivation of hiPS

NCT ID: NCT03898817

Last Updated: 2021-12-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

3 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-09-09

Study Completion Date

2017-09-09

Brief Summary

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Topic of this work is the involvement of replicative helicases in human premature ageing syndrome. Replicative helicases are ubiquitous and essential during numerous reactions of the DNA metabolism.

The family of replicative helicases (RecQL) is involved in the replication/repair of the DNA and in the telomere maintenance. There are 5 enzymes in human and 3 of them are involved in clinically recognizable syndromes: WRN for the Werner syndrome, BLM for the Bloom syndrome and RECQL4 for the Rothmund Thomson syndrome. All are responsive of a high cancer risk due to genomic instability. Molecular and cellular mechanisms involved in these diseases of ageing are unknown. Moreover, for all of them, there is not therapeutic or preventive solution.

Detailed Description

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For understanding the involved mechanisms we would like to model the 3 diseases with hiPS (human induced Pluripotent Stem cells) from somatic cells of patients. The patient recruitment was organized by the Montpellier and Nîmes public hospitals.

The project is to generate a hiPS cell line for the 3 syndromes from fibroblasts and/or blood samples. Then, we could induce differentiation of hiPS to a target cell line of the diseases. Finally we could study the disease development following the genomic instability (karyotype, array-CGH) and the cellular ageing (senescence-associated heterochromatin foci, telomere length).

For each mutated enzyme, we will perform a transcriptional profiling (splice, mRNA quantification) and protein studies (western blot). All results will be compared to wild type cells.

Conditions

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Age Problem

Keywords

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helicase induced pluripotent stem cells

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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Taking of cutaneous cells by biopsy

Taking of cutaneous cells by biopsy and a sample of blood

taking of cutaneous cells

Intervention Type OTHER

Taking of cutaneous cells by biopsy Sample of blood

Interventions

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taking of cutaneous cells

Taking of cutaneous cells by biopsy Sample of blood

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Patinet with one of the 3 helicase-associated precoce aging desease

Exclusion Criteria

* Minor and /or mentally incapable patient
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Montpellier

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Vincent GATINOIS, harmD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Montpellier

Locations

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University Hospital Montpellier

Montpellier, , France

Site Status

Countries

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France

Other Identifiers

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9360

Identifier Type: -

Identifier Source: org_study_id