AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

Tran, NT; Heiner, C; Weber, K; Weiand, M; Wilmot, D; Xie, J; Wang, D; Brown, A; Manokaran, S; Su, Q; Zapp, ML; Gao, GP; Tai, PWL

Gao, GP; Tai, PWL (corresponding author), Univ Massachusetts, Horae Gene Therapy Ctr, Med Sch, 386 Plantat St, Worcester, MA 01605 USA.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020; 18 (): 639

Abstract

The gene therapy field has been galvanized by two technologies that have revolutionized treating genetic diseases: vectors based on adeno-associated v......

Full Text Link