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Alessandra Recchia, PhD - "Modelling the CRISPR/Cas9 system to correct monogenic disorders"

Assistant Professor of Molecular Biology, Department of Life Sciences, University of Modena and Reggio Emilia, Italy
When Nov 18, 2019
from 12:00 PM to 01:00 PM
Where Tigem, Vesuvius Auditorium
Contact Name
Contact Phone 081-19230659
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Abstract
The CRISPR/Cas9 system is an effective genomic scissor guided by a short guide RNA to cleave specific DNA target sequences. The Cas9 introduces double strand breaks into the target gene, stimulating the cell’s DNA repair mechanisms of homology-directed repair and non-homologous end joining. Triggering these cellular mechanisms to purposely edit the gene of interest, CRISPR/Cas9 technology has earned a tremendous impact on genetic manipulation. For its simplicity, CRISPR/Cas9 has been studied and applied the most by scientists and clinicians and also proposed as a gene therapy tool for monogenic disorder. In this seminar I will present you our experience on CRISPR/Cas9 applications to tackle monogenic disorders. Employing different delivery systems and different SpCas9 variants, I will report correction of genetic defects ex vivo, in patients’ cell, and in vivo in preclinical model of dominant retinitis pigmentosa.

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