Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
Naoya Uchida, Linhong Li, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Juan J. Haro‐Mora, Selami Demirci, Alexis Leonard, Aylin Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Cornell Allen, Madhusudan V. Peshwa, Suk See De Ravin, Robert E. Donahue, Harry L. Malech, John F. Tisdale
Abstract
HSCs are engraftable in xenograft mice and non-human primates. These findings are helpful in designing HSC-targeted gene correction trials.
Topics & Concepts
Locus (genetics)CD34DiseasePrimateBiologyImmunologyCellGeneCancer researchMedicineStem cellCell biologyPathologyGeneticsNeuroscienceVirus-based gene therapy researchCRISPR and Genetic EngineeringRNA Interference and Gene Delivery