Article

Preclinical studies for plant-based oral enzyme replacement therapy (Oral-ERT) in Pompe disease knockout mice with transgenic tobacco seeds expressing human GAA (tobrhGAA)

2021-11-12

Abstract excerpt

<h4>ABSTRACT</h4> 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 onset). Our objective is to develop an innovative and affordable approach for enzyme replacement therapy (ERT) via oral administration (Oral-ERT) to maintain a sustained...

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Literature Corpus work: a526adfe-aff4-5e49-b1f2-53f9118b6835
DOI: 10.1101/2021.11.11.468227

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