Gene-editing technologies show clinical promise despite embryo research risks

Recent advancements and challenges in gene-editing technologies are highlighting both the therapeutic potential and the biological risks of genetic modification. Research from the Broad Institute shows that technologies such as CRISPR-Cas9, base editing, and prime editing are currently being utilized in over 25 clinical trials to address conditions including leukemia, rare genetic diseases, and high cholesterol.

Broad Institute's work, supported by NIH funding, has contributed to the development of tools for detecting cancer DNA in blood tests, the gnomAD genetic variant database, and datasets used to train Google DeepMind’s AlphaGenome AI model. Additionally, the FDA granted accelerated approval for a lung cancer drug developed using Broad Institute science.

However, a study from Columbia University researchers published in Nature indicates that while next-generation base editing is more precise than traditional CRISPR—acting more like a pencil than scissors—it still presents significant risks in human embryos. The research, led by Dieter Egli, suggests that these risks currently prevent the clinical use of such techniques in embryos, as scientists continue to study how early human development handles targeted genetic modifications.