Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim, J., C. Hu, C. Moufawad El Achkar, L. E. Black, J. Douville, A. Larson, M. K. Pendergast, et al. 2019. “Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease”. N Engl J Med 381: 1644-52.