CRISPR-mediated strategies for the treatment of laminopathies and orphan tumors

Ignacio PÉREZ DE CASTRO - Gene Therapy Unit, Institute of Rare Diseases Research, ISCIII (Majadahonda, Spain)

Invited by Yannick ARLOT


CRISPR-mediated gene edition is based on a bacterial pseudo-immune system. In 2013, this molecular tool was used to accomplish genome editing in mammalian cells. Since then, CRISPR has been applied to modify the genome of multiple cell lines and several species and, more importantly, to genetically cure diseases. In our group, we are using CRISPR-associated approaches to search for new therapeutic strategies for rare diseases. Specifically, we are focused on the congenital muscular dystrophy associated to LMNA (L-CMD) and the granulosa cell ovarian tumors. These diseases are characterized for the presence of a causal point mutation on the LMNA and FOXL2 genes, respectively. During my talk I will summarized the results we have obtained in these two research lines since we started with these projects one year ago.

>> Friday, June 9, at 11:00 - IGDR conference room (ground floor of Building 4 / Villejean Campus)

Seminar in English, free entry subject to availability

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