What was achieved: Researchers corrected SCN1A mutations in mice using prime or base editing, reducing seizures and improving survival. Why it matters: These are the first demonstrations of directly ...

In a world first, a research team at the University of Zurich has successfully treated mice carrying an inherited form of ...

A medical team treated the infant KJ for a rare genetic disorder using gene-editing therapy from February to April 2025, achieving a breakthrough that could help millions, according to two statements.

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of ...

A biotech company is looking to get approval next after their recent discovery.

DENVER, Jan. 28, 2026 (GLOBE NEWSWIRE) -- CRISPR gene-editing, and related genome engineering technologies, are reshaping industries from medicine to materials, with breakthroughs in treating genetic ...

The ethics of using safe gene therapies to improve the health and cognition of Down syndrome children and adults.

In May 2025, the world celebrated the success of KJ, an infant who was treated with the world’s first personalized CRISPR gene editing therapy. KJ was born with severe carbamoyl phosphate synthetase 1 ...

Please provide your email address to receive an email when new articles are posted on . In this video, Mark Sulkowski, MD, discusses clinical trials in gene editing therapy and basic studies in ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...

CRISPR functions as a programmable genetic memory system derived from bacterial immune defenses against viral infections. Guide RNA allows for rapid multiplexed targeting compared to older ...

A team of doctors and scientists has used a tailor-made gene-editing therapy to treat an infant with a rare genetic condition, a medical first that opens the door to a new era of individualized ...