Two pediatric deaths in Chinese CRISPR trials reignite debate over AAV delivery risks, as many of the 15 genetic-medicine experts Endpoints spoke with point to high viral doses rather than gene editin...

China’s cheap, fast and quiet system for testing cutting-edge medicines is facing scrutiny after the deaths of three people in investigator-initiated trials.

With a second delayed report of a child's death in China, criticism is rising of a looser form of clinical trial. One expert: what "bothers me is the lack of transparency."

Analysis of age-dependent allele frequency trajectories reveals 168 candidate variants under ongoing natural selection in Han Taiwanese individuals. Many are associated with disease susceptibility and...

Parents want accountability after study went disastrously wrong, Science and Retraction Watch investigation reveals

INSTALL overcomes fundamental challenges for DNA delivery and integration methods by synergizing immune-stealth nucleic acids with recombinases to enable kilobase-scale integration strategies without ...

The use of genome editing in early embryos has pulled back the curtain on the role of one of the key genes that orchestrates the first stages of human development, researchers reported.

A new report in the American Journal of Human Genetics describes a novel disorder caused by biallelic loss-of-function variants in the TMEM63B gene, which...

The authors report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders and other liver-centered disorders, as well as th...

The authors report initial proof-of-concept studies supporting a customizable prime editing platform geared to the treatment of 7 urea cycle disorders and other liver-centered disorders, as well as th...

Despite increasing focus on the intersection of genetic and social risk factors on health, heterogeneity in how these factors are measured, distributed, and analyzed across populations and datasets co...

When ancient humans mated, dad was a Neanderthal, mom was Homo sapiens.

Here, we offer a perspective on the US legal landscape regarding the promotion of competition, with specific attention given to the human genetics and genomics industry. We highlight current policy on...

A new FDA pathway meant to enable one-time gene-editing cures could, ethicists warn, become a Pandora's box.

Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance,…

Engineered deletions are a powerful probe for studying genome architecture, function, and regulation. Yet, the lack of effective methods to create them in large numbers and at multi-kilobase scale has...

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the approach.