How does the CRISPR Down Syndrome breakthrough work to remove the extra chromosome 21? We are closer than ever to a world where a genetic condition affecting one in every 700 babies could be addressed ...

Developing a gene therapy typically takes years, but when Baby KJ was diagnosed with a deadly genetic condition, scientists had only months. What followed was a successful collaboration among ...

The CRISPR-Cas gene-editing system has long been the focus of research as a promising tool in genome editing. However, the emphasis has been on its underlying mechanisms and nucleases. In contrast, ...

Casgevy sickle cell gene therapy is now FDA-approved for children as young as two, opening access for 5,500 more U.S. kids.