Preclinical studies for plant-based oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice with transgenic tobacco seeds expressing human GAA (tobrhGAA)
Frank T. Martiniuk, Adra Mack, Justin Martiniuk, Shoreh Miller, Gregory O. Voronin, David C Reimer, Nancy Rossi, Leslie Sheppard Bird, Sussan Saleh, Ruby Gupta, Mariel Nigro, Peter Meinke, Benedikt G. H. Schoser, Feng Chi Wu, Angelo Kambitsis, , Kam-Meng Tchou-Wong
This paper demonstrates that oral administration of ground transgenic tobacco seeds expressing human GAA (tobrhGAA) improves symptoms and enzyme activity in Pompe disease knockout mice.
bioRxiv · Nov 11, 2021 · PreprintGenetics