A new proof-of-concept method reliably engineered a hotspot mutation of SF3B1, a gene-splicing gene, into diverse cancer cell lines, outperforming other contemporary editing approaches.
Several years of preliminary research results suggest chimeric antigen receptor (CAR) T-cell therapy could benefit pediatric patients with refractory systemic lupus erythematosus (SLE).
The gene therapy landscape has changed dramatically in the past 5 years, and investigators wanted to capture and analyze the details of current and upcoming trials, along with their potential impact on patients.