Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

Gene-set analysis seeks to identify the biological mechanisms underlying groups of genes with shared functions. Large language models (LLMs) have shown promise in generating functional descriptions ...

A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

Researchers found that paired pathogenic variants can restore protein function rather than make it worse. This phenomenon ...

With a shared purpose, we can realize the true promise of CRISPR and improve healthcare, providing hope to more patients with ...

Sometimes, in genetics, two wrongs do make a right. A research team recently showed that two harmful genetic variants, when occurring together in a gene, can restore function—proving a decades-old ...

A new study confirms this long-held theory, discovering that 60 percent of defective genetic pairs impacting the production ...

Johns Hopkins researchers have developed a powerful new AI tool called Splam that can identify where splicing occurs in genes—an advance that could help scientists analyze genetic data with greater ...

Could a gene regulatory network in gut microbes have evolved its elaborate and tightly regulated molecular machinery only to pump out antibiotics indiscriminately? Researchers from the Institute of ...