CADASIL Clinical Trial
Official title:
Obtention d'un modèle Cellulaire de la Maladie CADASIL à Partir de Fibroblastes cutanés de Patients
Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leucoencephalopathy
(CADASIL) is an archetypal small vessel disease of the brain caused by dominant mutations in
the NOTCH3 receptor. Cardinal vascular lesions include deposition of granular osmiophilic
material (GOM) within the basal lamina of smooth muscle cells, progressive smooth muscle
cell loss, and fibrosis of the media. Pathogenic mutations alter the number of cysteine
residues in the extracellular domain of NOTCH3 (Notch3 ECD), leading to its abnormal
accumulation in the GOM deposits. Vascular smooth muscle cell has been identified as the
primary target cell in this disease. Pathophysiological processes leading from NOTCH3
mutations to smooth muscle cell loss remain poorly understood.
The investigators propose to study these mechanisms by reprogramming skin cells to become
stem cells and then differentiating them to vascular smooth muscle cells.
The hypothesis of this study is that the differentiated smooth muscle cells will display the
characteristic features of CADASIL, ie, Notch3 ECD accumulation and GOM deposits.
n/a
Observational Model: Case-Only, Time Perspective: Prospective
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