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Target intelligence / Profile preview
Gem nuclear organelle associated protein 5 (GEMIN5)
- Target
- GEMIN5
- Molecular classification
- WD repeat protein, RNA-binding protein, Component of the survival of motor neurons (SMN) complex, Other
01
Overview
Gem nuclear organelle associated protein 5 (GEMIN5) is a WD repeat protein that acts as a critical component of the survival of motor neurons (SMN) complex, which is essential for the assembly of small nuclear ribonucleoproteins (snRNPs) required for mRNA splicing in the cell[1][2][3][4]. GEMIN5 performs key roles in the recognition and delivery of snRNA to the SMN complex and is further involved in the regulation of translation by binding the 3'-UTR of SMN1 mRNA and interacting with ribosomes[3]. Structurally, GEMIN5 contains a WD40 repeat domain, a tetratricopeptide repeat (TPR)-like dimerization domain, and two non-conventional RNA-binding sites (RBS1 and RBS2)[2]. Mutations in GEMIN5 have been linked to neurodevelopmental disorders such as cerebellar atrophy, intellectual disability, and epilepsy, indicating its essential role in nervous system development and maintenance[2][8]. There is currently no evidence that GEMIN5 is considered a therapeutic target, nor are there known drugs that directly interact with or modulate this protein[3].
- Other names
- Gem-associated protein 5GEMIN5GEMIN-5gem (nuclear organelle) associated protein 5
02
Biological functions
Assembly of small nuclear ribonucleoproteins (snRNPs)mRNA splicingsnRNA recognition and deliveryRegulation of protein synthesisRibosome interactionmRNA translation regulation
03
Disease associations
Neurodevelopmental disorder with cerebellar atrophy and motor dysfunctionAutosomal recessive non-syndromic intellectual disabilityEpileptic encephalopathies (with biallelic variants)Other neurodevelopmental disorders
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