Mariana Duarte

Mariana Duarte is a Master's student within the project "LAMA2-MD: establishment of a new gene therapy strategy using patient- derived cell lines", under the supervision of Prof. Dra. Ana Rita Carlos , MMD. The project aims are constructing a unique CRISPR-Cas9 vector to correct mutations that lead to LAMA2-MD and maximizing its delivery to muscle cells by using highly muscle-specific rAAV vectors in patient-derived cells.