disease 2,677 words KG: ent-dise-b70d8e33 2026-03-23
kind:diseasesection:diseasesstate:published
Contents

C9orf72 Repeat Expansion in ALS

Disease Info
Endosomal traffickingProper sorting and transport of vesicles within cells
Lysosomal functionDegradation and recycling of cellular components
Neuronal transportMovement of organelles and signaling complexes along axons
Synaptic vesicle dynamicsRegulating neurotransmitter release at presynaptic terminals
SRSF2 and other splicing factorsLeading to widespread alternative splicing defects
Pur alphaA multi-functional RNA-binding protein critical for RNA transport
DatabasesOMIMOrphanetClinicalTrialsPubMed

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