Gene Editing: The Lessons of a Medical Breakthrough
Re 'Custom Gene-Editing Treatment Helps Baby in World's First Case' (front page, May 16):
Your article highlighting the remarkable work of Dr. Rebecca Ahrens-Nicklas in developing a bespoke gene-editing therapy for KJ, a child with a rare disorder, is a powerful testament to translational research that bridges the clinic and the lab. It is no coincidence that Dr. Ahrens-Nicklas is a physician-scientist trained in both medicine and research through a program funded by the National Institutes of Health.
Dr. Ahrens-Nicklas and I were classmates in the Tri-Institutional M.D.-Ph.D. Program, run jointly by Weill Cornell Medicine, Rockefeller University and Memorial Sloan Kettering Cancer Center. Our peers from this program are advancing our understanding of cancer, H.I.V.-AIDS and other illnesses, each drawing on the unique ability to connect patient care with scientific discovery.
These dual-degree programs exist to train precisely the kind of visionary thinkers who can identify unmet clinical needs and then return to the lab to devise novel solutions. This is possible only when scientists understand disease at both the molecular and human level.
Recent and proposed cuts to the National Institutes of Health threaten the pipeline that makes such breakthroughs possible. Without strong federal support, we risk losing a generation of physician-scientists — and with them, the kinds of lifesaving advances described in this incredible story.
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