Please fill out this form to register for the upcoming Webinar: Non-Viral Ex Vivo Gene Editing of T Cells and HSCs for Cell Therapy  presented by Dr. Samuele Ferrari, Project Leader, Dr. Luigi Naldini’s Lab, SR-TIGET.

Date: Wednesday, October 4, 2023

Time: 7:00am PT | 10:00am ET | 4:00pm CET

Abstract

Ex vivo gene editing of T cell and hematopoietic stem and progenitor cells (HSPCs) holds tremendous promise for treating diseases. Gene editing encompasses delivery of a programmable editor RNA or ribonucleoprotein, often achieved ex vivo by electroporation. Lipid nanoparticles are actively being investigated to address the various shortcoming of electroporation to provide a clinically validated alternative to researchers and cell therapy developers.  

In this webinar, we demonstrate that:

•  Electroporation can be the main culprit of cytotoxicity in T cells causing cell death and cell cycle delay using multi-omics analysis
•  Electroporation can decrease the clonogenic potential of human HSPCs in vitro
•  Nuclear RNA-LNPs nearly abolished cell death and ameliorated cell growth, improving tolerance and yielding higher number of edited cells compared to electroporation
•  LNPs can allow for versatile, efficient and harmless ex vivo gene editing in hematopoietic cells for the treatment of human disease