The first phase of the project led by APTEEUS™ in collaboration with the charity Les Petits Mec P2 is drawing to a close. A duplication of the MECP2 gene leads to the overexpression of the MeCP2 protein, which is present in all the body’s cells. APTEEUS™ has chosen to use fibroblasts derived from patients’ skin to demonstrate the overexpression of the protein and to attempt to regulate it using molecules already in use for other indications.
Out of more than 5,000 molecules tested in a screening programme, APTEEUS™ identified several molecules capable of reducing the levels of the MeCP2 protein in the cells of two patients from the association.
APTEEUS™ is currently collecting data on each of the selected molecules so that we can assess their medical value in consultation with doctors.
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