Dr Ayal Hendel from The Mina and Everard Goodman Faculty of Life Sciences, Nanotechnology Institute, at Bar-Ilan University (Israel) is giving a seminar on July 1st at 1 pm at Imagine Institute.
He is an expert in CRISPR/Cas9 and editing of hematopoietic cells. Please find below the summary of his presentaion:
Genome editing of human hematopoietic stem and progenitor cells holds a great therapeutic potential. Our research focuses on developing novel genome editing strategies as curative therapy for gene therapy of hematopoietic genetic disorders such as severe combined immunodeficiency (SCID). CRISPR genome editing requires delivery of both the Cas9 nuclease and the targeting guide RNA (gRNA). The gRNA component can be generated in multiple ways, each with advantages and disadvantages. The use of synthetic gRNA allows for the incorporation of chemical modifications for enhanced properties including improved stability. Here we present gene editing results using chemically modified synthetic single guide RNA (sgRNA) molecules and chemically modified crRNA:tracrRNA molecules (two separate strands to form a complete guide RNA) delivered as a ribonucleoprotein (RNP) complex. We show that chemical alterations to synthesized guide RNAs enhance genome editing efficiency in human hematopoietic stem and progenitor cells in a locus-specific manner. In addition, we describe an approach for unbiased identification of potential gene-specific off-target effects (OTEs) by GUIDE-seq, and their subsequent quantification by the rhAmpSeq technique (a multiplexed, PCR-based targeted enrichment approach). This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of therapeutic applications for other primary hematopoietic cells.
For more info about Dr Hendel laboratory, visit his website: https://www.hendelab.com/
Location : Imagine Institute – 24 boulevard du Montparnasse – 75015 Paris