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 ...

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 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, ...

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) ...

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 ...

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. Researchers described the case in a new study, ...

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 ...

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with ...

A landmark Huntington's trial slows disease progression by 75%, signalling how RNA- and CRISPR-based tools redefine treatment for rare genetic disorders. Significant advancements in gene silencing and ...