Drug screens of NGLY1 deficiency in worm and fly models reveal catecholamine, NRF2 and anti-inflammatory-pathway activation as potential clinical approaches

• Main Authors: Sangeetha Iyer (Author), Joshua D. Mast (Author), Hillary Tsang (Author), Tamy P. Rodriguez (Author), Nina DiPrimio (Author), Madeleine Prangley (Author), Feba S. Sam (Author), Zachary Parton (Author), Ethan O. Perlstein (Author)
• Format: Book
• Published: The Company of Biologists, 2019-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

N-glycanase 1 (NGLY1) deficiency is an ultra-rare and complex monogenic glycosylation disorder that affects fewer than 40 patients globally. NGLY1 deficiency has been studied in model organisms such as yeast, worms, flies and mice. Proteasomal and mitochondrial homeostasis gene networks are controll...