Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

per Kim, J., Hu, C., Moufawad El Achkar, C., Black, L.E., Douville, J., Larson, A., Pendergast, M.K., Goldkind, S.F., Lee, E.A., Kuniholm, A., Soucy, A., Vaze, J., Belur, N.R., Fredriksen, K., Stojkovska, I., Tsytsykova, A., Armant, M., DiDonato, R.L., Choi, J., Cornelissen, L., Pereira, L.M., Augustine, E.F., Genetti, C.A., Dies, K., Barton, B., Williams, L., Goodlett, B.D., Riley, B.L., Pasternak, A., Berry, E.R., Pflock, K.A., Chu, S., Reed, C., Tyndall, K., Agrawal, P.B., Beggs, A.H., Grant, P.E., Urion, D.K., Snyder, R.O., Waisbren, S.E., Poduri, A., Park, P.J., Patterson, A., Biffi, A., Mazzulli, J.R., Bodamer, O., Berde, C.B., Yu, T.W.
Publicat a N Engl J Med (2019)