Drug repositioning study at APTEEUS™

The first phase of the project conducted by APTEEUS™ with Les petits Mec P2 Association is coming to an end. Duplication of the MECP2 gene results in overexpression of the MeCP2 protein present in all cells of the body. APTEEUS™ chose to use fibroblasts from the patients' skin to highlight the overexpression of the protein and attempt to regulate it with molecules already used for other indications.

Among more than 5,000 molecules tested during the screening, APTEEUS™ has identified several molecules capable of reducing the amount of MeCP2 protein in the cells of 2 patients in the association.

APTEEUS™ is currently collecting data on each of the selected molecules so that we can judge their medical interest with physicians.

Illustration of screening results. The control level of MeCP2 is normally less than 6.0.105, whereas the patient cells have a level close to 1,0.106.

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Aleix and his parents talk about their life with the syndrome

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