Evaluation of two in vitro assays for tumorigenicity assessment of CRISPR-Cas9 genome-edited cells
Myriam Lemmens, Benoit Fischer, Michael Zogg, Lindsey Ulkus Rodrigues, Gráinne Kerr, Alberto Del Río-Espínola, Fanny Schaeffer, Danilo Maddalo, Valérie Dubost, Alessandro Piaia, Arne Mueller, Ulla Plappert‐Helbig, Ulrike Naumann, Jasmin Haegele, Alex Odermatt, Hans‐Jörg Martus, Silvana Libertini
Abstract
tumorigenicity study failed to detect the known tumorigenic potential of PTPN12 deletion, demonstrating the relevance of GILA and SACF in tumorigenicity testing. In conclusion, SACF and GILA are both attractive and valuable additions to preclinical safety assessment of gene therapy products.
Topics & Concepts
CRISPRCas9In vitroIn vivoGenome editingGenetic enhancementGeneTransformation (genetics)GenomeBiologyCell cultureCancer researchComputational biologyMolecular biologyGeneticsCRISPR and Genetic EngineeringVirus-based gene therapy researchCAR-T cell therapy research