Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...

KJ Muldoon, a 10-month-old baby, was diagnosed with the genetic disease carbamoyl-phosphate synthetase 1 deficiency after he ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...