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This study examines the collection of cell samples through skin biopsy in order to develop and validate a gene therapy for Huntington's disease. It is enrolling people aged 18-70 who carry a CAG≥36 allele in the HTT gene. It is being run as a study with no assigned phase and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * 18 ≤ age ≤ 70 years. * Signed written, free and informed consent to participate in the study. * Patients with a CAG≥36 allele (with reduced or full penetrance). penetrance) * People affiliated to or benefiting from a social security scheme. Exclusion Criteria: * Individuals who have participated in a gene therapy trial using AAV, ASO, mi/si/shRNA administration, likely to disrupt expression, splicing of pre-mRNAs, mRNA splicing, mRNA expression/regulation/translation, energy or protein metabolism directly or indirectly linked to the Huntingtin gene (HTT), its transcripts and proteins. * Clinical or paraclinical elements that may suggest a differential diagnosis. * People unable to express their consent. * Pregnant, breast-feeding or parturient women * People deprived of liberty by administrative or judicial decision * People under legal protection (curatorship, guardianship).