I do not like the Trump administration's attempts to destroy and cut NIH research funding in the United States. Why? Because without it, advances like this in medicine do not happen. At least they will not happen in the United States. Direct gene editing used to save a baby in PA from a metabolic condition that shortens the lives of any infant who has it. The condition lacks an enzyme to help control the buildup of ammonia from protein in the diet. Without some protein, we also perish. When I came to this country as a scientist, I took on my biggest client, the United States of America, the number one research driver in the world. I want to see my client win its case. It is simple.
Read about the first patient, a baby, treated with personalized CRISPR gene editing therapy at CHOP. This study was supported by grants from the National Institutes of Health Somatic Cell Genome Editing Program (U01TR005355, U19NS132301), as well as additional National Institutes of Health grants (R35HL145203, U19NS132303, DP2CA281401, P01HL142494). In-kind contributions were made by Acuitas Therapeutics, Integrated DNA Technologies, Aldevron, and Danaher Corporation. Additional funding was provided by the CHOP Research Institute’s Gene Therapy for Inherited Metabolic Disorders Frontier Program.
The paper in the New England Journal of Medicine is here: Musunuru et al, “Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.” N Engl J Med. Online May 15, 2025.
