Customized gene-editing technology shows potential to treat lethal pediatric disease

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood. Currently, there is no effective treatment or ... Read More

The Next Era of Gene Editing Will Be Disease Agnostic

At the WIRED Health summit, biochemist David Liu said his lab is on the verge of revealing a new gene-editing technique that could target multiple unrelated diseases. Read More

Scientists develop technology that brings new precision to genome editing

The FDA's approval of the first CRISPR-Cas9–based gene therapy marked a major milestone in biomedicine, validating genome editing as a promising treatment strategy for disorders like sickle cell ... Read More

Gene discovery could lead to new treatments for cancer and other diseases tied to vitamin D deficiency

Vitamin D plays a vital role in the human body, far beyond just keeping bones strong. It helps your muscles move, allows your nerves to carry messages, supports your immune system, and helps control ... Read More

High-Precision Base Editing Clinical Treatment for Sickle Cell Disease — CorrectSequence Therapeutics' CS-101 Achieves Promising Results in First Patient

SHANGHAI, Aug. 26, 2025 /PRNewswire/ -- CorrectSequence Therapeutics Co., Ltd. (Correctseq), a clinical-stage biotechnology company pioneering transformer Base Editing (tBE) technology for the ... Read More

Gene editing technology could be used to save species on the brink of extinction

Earth's biodiversity is in crisis. An imminent "sixth mass extinction" threatens beloved and important wildlife. It also threatens to reduce the amount of genetic diversity—or variation—within species ... Read More

Gene editing therapy exa-cel improves quality of life in sickle cell disease and beta thalassemia

"This is the first time we've been able to measure improvements in quality of life after treatment with gene editing technology," said Josu de la Fuente, PhD, director of the Paediatric Blood and ... Read More

Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to Develop Gene Editing Therapy

BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of a gene editing therapy ... Read More

The quest to create gene-edited babies gets a reboot

A Chinese scientist horrified the world in 2018 when he revealed he had secretly engineered the birth of the world's first gene-edited babies. His work was reviled as reckless and unethical because, ... Read More