Researchers have found that a new base-editing gene therapy can help treat a rare neurodevelopmental disorder called Snijders Blok–Campeau syndrome caused by mutations in the CHD3 gene. A specialized ...

Researchers have engineered a new class of adenine base editors that reduce unwanted bystander mutations by two to three times compared to the widely used ABE8e, while preserving the editing power ...

Microbiome research has faced a hurdle from the get-go: the inability to edit the microbial genome in vivo. Until now, bacterial genomes had to be modified outside—and reintroduced into—the host ...

Scientists at St. Jude Children’s Research Hospital report that they have developed an unbiased, sensitive, and resource-efficient method to identify small, off-target sites that pose a safety risk ...

A Beam Therapeutics gene-editing therapy designed to fix a mutation at the root of a rare liver protein deficiency now has early signs of efficacy along with safety data that ease some concerns about ...

Forbes contributors publish independent expert analyses and insights. Juergen Eckhardt leads Bayer’s impact investment unit, Leaps by Bayer. Dr. David Liu, pictured with former lab members Holly Rees ...

Editor’s note: On June 3, 2025, KJ Muldoon was discharged from the hospital and is now at home with his family. When a baby born in Philadelphia was announced as the first person to get a gene therapy ...

(L) Corresponding author Shengdar Tsai, PhD, and (R) co-first author Varun Katta, both of the St. Jude Department of Hematology. While traditional genome editing technology uses CRISPR-Cas9 to cut a ...

Engineers at the University of Pennsylvania and Rice University have refined a technology for editing individual genetic "base pairs" to a new level of precision, opening the door to safer, more ...

A single mutation in a person’s genome can be the difference between health and disease, and with base editing, researchers can recreate these mutations in a laboratory to study how genetic changes ...