Commenting on Cargill’s article, this Commentary examines how gene therapy research is regulated in the United States and how oversight of the field has developed. It discusses recent applications of gene therapy technologies and their implications for oversight, and of the impact of ordered cuts to NIH-funded research on gene therapy developments more broadly. Ultimately, it underscores the need for adaptive oversight frameworks for research involving emerging biotechnologies that balance scientific innovation, safety, and ethical considerations, and for effective public engagement on the acceptable use of these technologies, notwithstanding the discontinuation of NIH’s advisory mechanism established for this purpose.