In a landmark advancement for genetic medicine, a novel gene-editing therapy known as exagamglogene autotemcel, or exa-cel, has been approved for treating sickle cell disease (SCD) in both the United States and the United Kingdom. This therapy, marketed as Casgevy, utilizes CRISPR-Cas9 technology to modify patients' own stem cells, aiming to alleviate the debilitating effects of SCD.

A once-fatal condition in infants now has a promising treatment thanks to a cutting-edge gene therapy called onasemnogene abeparvovec.

A new type of cancer treatment may be on the horizon—and it comes from an unlikely source: a virus. Researchers have found that a tiny part of a virus, called 3C protease, could be a powerful weapon against many types of solid tumors, which are cancers that form lumps in organs like the lungs, liver, or breast.

Cystic fibrosis is primarily caused by the F508del mutation in the CFTR gene, leading to defective chloride ion transport in epithelial cells. Traditional treatments require lifelong administration of small-molecule drugs, which, while beneficial, do not offer a permanent solution. Prime editing (PE) emerges as a promising gene-editing tool capable of precise DNA modifications without inducing double-stranded breaks.