The case for germline gene correction: state of the science.

Germline gene editing refers to altering the DNA in the reproductive cells (gametes or embryos). Germline gene editing experiments in human embryos have primarily focused on correcting genetic mutations linked to inherited diseases. This technology has the potential to prevent genetic disease before birth and in future generations. Advances in CRISPR-Cas9 and other gene-editing tools have accelerated scientific progress, raising both promise and safety and ethical concerns. A translational pathway for human heritable genome editing will require an approach that integrates scientific validation, ethical oversight, regulatory frameworks, and public engagement.