Over the past decade, the field of human embryo editing has witnessed remarkable advancements and triggered significant ethical debates. Since the first reported editing in 2015, the field has shown promise for treating genetic disorders and studying early development, the field has shown promise for treating genetic disorders and studying early development, but it still grapples with safety concerns, including off-target effects, mosaicism and the long-term impacts of genetic alterations, as well as ongoing ethical controversies.
This review systematically synthesizes technological progress, key scientific hurdles, and ethical issues, while evaluating the potential applications and limitations of human embryo genome editing in disease treatment and basic research.
A comprehensive literature search was conducted using the electronic databases PubMed and Google Scholar, with the search period extending up to December 2025. Keywords including "human embryo genome editing," "germline genome editing," and "CRISPR/Cas9" were employed, either individually or in combination. In addition, both forward and backward citation tracking was performed on key articles to identify further relevant publications.
Key findings:
- Technologies including CRISPR/Cas9, base editors, and mitochondrial editing tools have demonstrated significant progress in correcting pathogenic mutations and enabling functional research in embryos.
- Substantial scientific challenges, including mosaicism, off-target effects, and chromosomal instability, remain critical barriers to clinical translation.
- The technology raises profound questions regarding heritable modifications, societal consensus, and the potential misuse, highlighted by the He Jiankui case.
- Responsible advancement will require improved editing accuracy, rigorous safety assessments, and internationally accepted ethical and regulatory standards.
This review provides a systematic and comprehensive overview of human embryo gene editing, offering a scientific foundation along with an analysis of the technical challenges and ethical considerations that will shape the future clinical translation of heritable genome editing.
The primary gene-editing tools, key advancements, and major technological barriers impeding progress in human genome editing.
DOI：10.1093/procel/pwag030