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…·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.