Gene Therapy in Patients with the Crigler–Najjar Syndrome
Lorenzo D’Antiga, M.D., Ulrich Beuers, M.D., Giuseppe Ronzitti, Ph.D., Nicola Brunetti-Pierri, M.D., Ulrich Baumann, M.D., Angelo Di Giorgio, M.D., Sem Aronson, Ph.D., Aurelie Hubert, Ph.D., Roberta Romano, M.D., Norman Junge, M.D., Piter Bosma, Ph.D., Giulia Bortolussi, Ph.D., Andrés F. Muro, Ph.D., Ravaka F. Soumoudronga, M.D., Philippe Veron, Ph.D., Fanny Collaud, Ph.D., Nathalie Knuchel-Legendre, M.A.Sc., Philippe Labrune, M.D., and Federico Mingozzi, Ph.D.
N Engl J Med 2023; 389:620-631 DOI: 10.1056/NEJMoa2214084 August 17, 2023
https://www.nejm.org/doi/full/10.1056/NEJMoa2214084?query=featured_home