Fig. 1
From: Reversibility of motor dysfunction in the rat model of NGLY1 deficiency
Protein expression and enzymatic activity of hNGLY1 in AAV9-hNGLY1-injected Ngly1−/− rats. Protein expression and enzymatic activity of hNGLY1 were restored in the CNS after intracerebroventricular injection of AAV9-hNGLY1 into Ngly1−/− rats. a The single-stranded AAV9.hNGLY1 construct. ITR; inverted terminal repeat, CMV; cytomegalovirus enhancer, polyA; human growth hormone polyadenylation signal, NGLY1; cDNA of human NGLY1 without 3’UTR. b Western blot of endogenous and exogenous NGLY1 protein levels in AAV-injected rats. c Enzymatic activity of NGLY1 proteins in the brains of AAV-injected rats (n = 1). WT; wild type rats, KO; Ngly1−/− rats. hGH poly(A) signal. Human growth hormone polyadenylation signal