RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …

CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat
disease. However, standard computational and biochemical methods to predict off-target …

CRISPR-Cas technology has rapidly changed life science research and human medicine.
The ability to add, remove, or edit human DNA sequences has transformative potential for …

Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits
relatively poor editing efficiency, restricting its overall utility. Here we isolate an engineered …

CRISPR-Cas is a versatile genome editing technology that has been broadly applied in both
basic research and translation medicine. Ever since its discovery, the bacterial derived …

CRISPR/Cas systems are novel gene editing tools with tremendous capacity and accuracy
for gene editing and hold great potential for therapeutic genetic manipulation. However, the …

Genome editing produces genetic modifications in somatic cells, offering novel curative
possibilities for sickle cell disease and β-thalassemia. These opportunities leverage clinical …

A growing number of gene therapy‐and gene editing‐based treatments for patients with
sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the …

Gene editing technologies such as zinc finger nuclease (ZFN), transcription activator-like
effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats …