Frank Martiniuk

No ORCID on file · 1 paper in corpus · active 2021
2021
Preclinical studies for plant-based oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice with transgenic tobacco seeds expressing human GAA (tobrhGAA)
·doi:10.1101/2021.11.11.468227

ABSTRACT Genetic deficiency of acid α -glucosidase (GAA) results in glycogen storage disease type II (GSDII) or Pompe disease (PD) encompassing at least four clinical subtypes of varying severity (infantile; childhood, juvenile and late o…