Scientists say they've put together a molecular toolkit that could be used to treat a variety of brain diseases, possibly ...

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

He’s among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his ...

KJ received the bespoke CRISPR treatment 6.5 months after birth, receiving three infusions of the therapy as of April 2025.

A mother has chosen to return to her home country with her 11-year-old daughter, who has a rare genetic disorder, after being ...

Leanna Scaglione was among the nearly 30,000 people who celebrated as they crossed the finish line of the RBC Brooklyn Half ...

Evidence is mounting that clinicians can treat serious genetic disorders prenatally by injecting medicine into the amniotic fluid, thus preventing damage that begins in utero.

The heartbreaking yet inspiring story of a 5-month old girl battling an extraordinarily rare genetic condition.

The Haenyeo’s unique traits stem from both genetics and lifelong practice, offering clues for improving cardiovascular health ...

A nonprofit called the n-Lorem Foundation is working on developing an individualized treatment that targets the genetic cause of rare diseases.

As luck would have it, though, researchers at CHOP and Penn Medicine had been trying to rapidly develop customized gene therapies for people with rare genetic diseases. And KJ seemed like the ...

"Understanding gene expression patterns could help develop new drugs or improve therapies," note the authors of the finding, ...