NCT03855631View on ClinicalTrials.gov

Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders

CompletedOBSERVATIONAL
Enrollment

8

Participants

Timeline

Start Date

September 28, 2020

Primary Completion Date

November 27, 2020

Study Completion Date

November 27, 2020

Conditions
Kabuki Syndrome 1
Interventions
GENETIC

Intervention on primary cultured cells

The intervention includes primary cultured cells, reprograming them into mesenchymal stem cells and CRISPR/Case9 gene therapy treatment on patients' cells

Trial Locations (1)

34295

Arnaud de villeneuve Hospital, Montpellier

All Listed Sponsors
collaborator

Association Française contre les Myopathies Telethon

UNKNOWN

lead

University Hospital, Montpellier

OTHER

NCT03855631 - Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders | Biotech Hunter | Biotech Hunter