CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters

por McDougald, Devin S., Duong, Thu T., Palozola, Katherine C., Marsh, Anson, Papp, Tyler E., Mills, Jason A., Zhou, Shangzhen, Bennett, Jean
Publicado 2019

Identification of a novel pathogenic missense mutation in PRPF31 using whole exome sequencing: a case report

por Bryant, Laura, Lozynska, Olga, Marsh, Anson, Papp, Tyler E, van Gorder, Lucas, Serrano, Leona W, Gai, Xiaowu, Maguire, Albert M, Aleman, Tomas S, Bennett, Jean
Publicado 2019