A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. Researchers described the case in a new study, ...

KJ Muldoon was diagnosed with a rare genetic disorder when he was born called severe CPS1 deficiency. KJ is one in a million babies that are diagnosed with this illness. Researchers and doctors used ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

In 2023, the first CRISPR–(Cas9)-based product was approved by the US Food and Drug Administration (FDA): Vertex/CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) for sickle cell disease (SCD) ...

An international scientific team says gene editing technologies can be repurposed to provide a transformative solution for restoring genetic diversity and saving endangered species. In an article ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. ‘Memory manipulation is inevitable’: How rewriting ...

Targeted DNA editing by CRISPR technology has great potential for applications in biotechnology and gene therapy. However, precise gene editing remains a challenge largely due to insufficient control ...

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...

A major medical milestone took place in May 2025, when doctors at the Children’s Hospital of Philadelphia used CRISPR-based gene editing to treat a child with a rare genetic disorder. Unlike earlier ...

A Bay Area biochemist has launched a new company to further research on gene editing on human embryos. Lucas Harrington said the research will focus on using the technology to prevent genetic disease ...

Gabriella Ngang, 18, of Farmington Hills is one of just three people in Michigan to take part in a clinical trial testing a new gene-editing therapy with the potential to cure her sickle cell disease.