Gene Therapy

NOV 3, 2026 · JOURNAL ARTICLE

Genotoxic safety and long-term engraftment dynamics of efficient translational CRISPR/Cas9 therapeutics for β-haemoglobinopathies from bench to clinical trials

Gene Therapy

CRISPR/Cas9-mediated gene editing targeting the BCL11A erythroid enhancer has transformed therapeutic approaches for transfusion-dependent β-thalassemia (β-thal) and sickle cell di

JUL 29, 2026 · JOURNAL ARTICLE

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study

Gene Therapy

Multi-site assay validation study establishing reproducibility of in vitro transformation assays in a single cell line model; supports exploratory use but does not yet establish clinical utility or predict in vivo tumorigenicity risk in actual CRISPR therapies.