PMID-sentid Pub_year Sent_text comp_official_name comp_offsetprotein_name organism prot_offset 34592985-8 2021 NPC1-/- i3Neurons recapitulate cardinal cellular NPC1 pathological features including perinuclear endolysosomal storage of unesterified cholesterol, accumulation of GM2 and GM3 gangliosides, mitochondrial dysfunction, and impaired axonal lysosomal transport. G(M3) Ganglioside 173-189 NPC intracellular cholesterol transporter 1 Homo sapiens 0-4 23881911-8 2013 Intervention with miglustat in NPC1 patients was accompanied by striking alterations in plasma (reductions in GM1 and GM3 gangliosides) and cerebrospinal fluid (CSF) (increased MCs) sphingolipids. G(M3) Ganglioside 118-134 NPC intracellular cholesterol transporter 1 Homo sapiens 31-35