Betibeglogene autotemcel gene therapy for non–β⁰/β⁰ genotype β-thalassemia

by Franco Locatelli, Alexis A. Thompson, Janet L. Kwiatkowski, John B. Porter, Adrian J. Thrasher, Suradej Hongeng, Martin G. Sauer, Isabelle Thuret, Ashutosh Lal, Mattia Algeri, Jennifer Schneiderman, Timothy S. Olson, Ben Carpenter, Persis J. Amrolia, Usanarat Anurathapan, Axel Schambach, Christian Chabannon, Manfred Schmidt, Ivan Labik, Heidi Elliot, Ruiting Guo, Mohammed Asmal, Richard A. Colvin, Mark C. Walters
Published 2022

Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

by C. Li, N. Narkbunnam, R. J. Samulski, A. Asokan, G. Hu, L. J. Jacobson, M. J. Manco-Johnson, P. E. Monahan, Marilyn J. Manco-Johnson, Brenda Riske, Ray Kilcoyne, Michael L. Manco-Johnson, Sharon Funk, Linda Jacobson, J. David Ingram, Thomas C. Abshire, Amy D. Shapiro, Michele R. Hacker, Leonard A. Valentino, W. Keith Hoots, Deborah Brown, George R. Buchanan, Donna DiMichele, Michael Recht, Cindy Leissinger, Shirley Bleak, Alan Cohen, Prasad Mathew, Alison Matsunaga, Desiree Medeiros, Diane Nugent, Gregory A. Thomas, Alexis A. Thompson, Kevin McRedmond, J. Michael Soucie, Harlan Austin, Bruce L. Evatt
Published 2018