CRISPR-Cas9-mediated homology-directed repair (HDR) is a versatile platform for creating
precise site-specific DNA insertions, deletions, and substitutions. These precise edits are …

Ex vivo gene editing in hematopoietic stem and progenitor cells (HSPCs) represents a
promising curative treatment strategy for monogenic blood disorders. Gene editing using the …

Abstract The DNA-PKcs inhibitor AZD7648 enhances CRISPR–Cas9-directed homology-
directed repair efficiencies, with potential for clinical utility, but its possible on-target …

Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

Sickle cell disease is a devastating blood disorder that originates from a single point
mutation in the HBB gene coding for hemoglobin. Here, we develop a GMP-compatible …

DNA double-strand breaks (DSBs) are repaired by a hierarchically regulated network of
pathways. Factors influencing the choice of particular repair pathways, however remain …

DNA repair in mammalian cells involves the coordinated action of a range of complex
cellular repair machinery. Our understanding of these DNA repair processes has advanced …

Mesanophrys sp. represents a parasitic ciliate, significantly jeopardizing the survival of
farmed swimming crabs (Portunus trituberculatus). However, certain drawbacks exist in …

This review critically examines the evolving landscape of chimeric antigen receptor (CAR) T-
cell therapy in treating solid tumors, with a particular focus on the metabolic challenges …

Augmenting cells with novel, genetically encoded functions will support therapies that
expand beyond natural capacity for immune surveillance and tissue regeneration. However …