Base editing and prime editing are advanced gene-editing technologies derived from the CRISPR/Cas9 system. Base editing allows precise conversion of one DNA base into another without causing double-stranded breaks, making it ideal for correcting point mutations. Prime editing offers even greater versatility by enabling targeted insertions, deletions, and all 12 possible base-to-base conversions. By avoiding double-stranded breaks, these methods reduce the risk of unintended mutations, enhancing the safety and efficacy of gene therapies. These technologies hold significant promise for HSPC gene therapy, as they can correct genetic defects at their source within HSCs, potentially providing long-lasting treatments for blood disorders like sickle cell disease and β-thalassemia.
The Genovese Lab is exploiting the most advanced HSPC culture and gene delivery strategies to maximize the efficiency and safety of base and prime editing to develop innovative therapeutic approaches. |
Base Editing of Hematopoietic Stem CellsKomor A.C. et al., Nature 2016; Gaudelli N.M. et al., Nature 2017
Prime Editing of Hematopoietic Stem CellsAnzalone A.V. et al., Nature 201
|