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GeneE
10 sources retrieved · Most recent: April 2026 · Index updated 14 days ago
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SIL1
SIL1 nucleotide exchange factor
Chromosome 5 · 5q31.2
NCBI Gene: 64374Ensembl: ENSG00000120725.14HGNC: HGNC:24624UniProt: A0A0S2Z6B4
77PubMed Papers
21Diseases
0Drugs
35Pathogenic Variants
FUNCTIONAL ROLE
Hub GeneTransporter
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
adenyl-nucleotide exchange factor activityprotein bindingidentical protein bindingGO:0005615Marinesco-Sjögren syndromeMarinesco-Sjogren syndromecataractGeneralized hypotonia
✦AI Summary

SIL1 is a nucleotide exchange factor that plays a critical role in endoplasmic reticulum (ER) protein homeostasis by regulating the HSP70 chaperone BiP (HSPA5) 1. SIL1 functions alongside GRP170 as one of two nucleotide exchange factors that facilitate BiP's ATP-ADP cycle, enabling proper protein folding and translocation in the ER lumen 23. The protein is essential for maintaining ER homeostasis through its regulation of BiP's chaperone activity in protein quality control pathways 1. Loss-of-function mutations in SIL1 cause Marinesco-Sjögren syndrome (MSS), a rare autosomal recessive multisystem disorder characterized by congenital cataracts, cerebellar ataxia, and vacuolar myopathy 1. Recent research has expanded the clinical spectrum of SIL1 deficiency to include peripheral nervous system abnormalities, with studies showing degenerative changes in peripheral nerves and neuromuscular junctions in both animal models and MSS patients 4. Additionally, SIL1 appears to have neuroprotective functions, as overexpression has been shown to improve cognitive impairment in Alzheimer's disease models by regulating amyloid precursor protein processing 5. The protein's involvement in neurological pathways is further supported by genome-wide association studies linking SIL1 variants to chr5 cough phenotypes 6.

Sources cited
1
SIL1 is a nucleotide exchange factor for BiP and loss of function causes Marinesco-Sjögren syndrome
PMID: 33557244
2
SIL1 and GRP170 are the two nucleotide exchange factors that regulate BiP's ATPase cycle
PMID: 25698114
3
SIL1 belongs to the HspBP1/Sil1 family of nucleotide exchange factors for Hsp70 chaperones
PMID: 25487014
4
SIL1 deficiency causes degenerative changes in peripheral nerves and neuromuscular junctions
PMID: 30468864
5
SIL1 overexpression improves cognitive impairment by regulating amyloid precursor protein processing
PMID: 39004862
6
SIL1 variants are associated with chronic cough phenotypes, implicating neurological pathways
PMID: 40675770
Disease Associationsⓘ21
Marinesco-Sjögren syndromeOpen Targets
0.80Strong
Marinesco-Sjogren syndromeOpen Targets
0.75Strong
cataractOpen Targets
0.42Moderate
Generalized hypotoniaOpen Targets
0.42Moderate
atrial fibrillationOpen Targets
0.38Weak
smoking initiationOpen Targets
0.36Weak
Cerebellar atrophyOpen Targets
0.34Weak
Global developmental delayOpen Targets
0.33Weak
apraxiaOpen Targets
0.33Weak
ConstipationOpen Targets
0.33Weak
ptosisOpen Targets
0.33Weak
StrabismusOpen Targets
0.33Weak
Urinary incontinenceOpen Targets
0.33Weak
lagophthalmosOpen Targets
0.24Weak
preeclampsiaOpen Targets
0.24Weak
Alzheimer diseaseOpen Targets
0.20Weak
genetic disorderOpen Targets
0.19Weak
ovarian dysfunctionOpen Targets
0.17Weak
neurodegenerative diseaseOpen Targets
0.14Weak
Spinocerebellar ataxia type 41Open Targets
0.09Suggestive
Marinesco-Sjoegren syndromeUniProt
Pathogenic Variants35
NM_022464.5(SIL1):c.1030-9G>APathogenic
not provided|Marinesco-Sjögren syndrome|See cases
★★☆☆2026
NM_022464.5(SIL1):c.1117del (p.Leu373fs)Pathogenic
Marinesco-Sjögren syndrome
★★☆☆2025→ Residue 373
NM_022464.5(SIL1):c.460C>T (p.Gln154Ter)Pathogenic
not provided|Marinesco-Sjögren syndrome
★★☆☆2025→ Residue 154
NM_022464.5(SIL1):c.645+2T>CPathogenic
Marinesco-Sjögren syndrome|not provided
★★☆☆2025
NM_022464.5(SIL1):c.936dup (p.Leu313fs)Pathogenic
Marinesco-Sjögren syndrome
★★☆☆2025→ Residue 313
NM_022464.5(SIL1):c.947dup (p.Arg317fs)Pathogenic
Marinesco-Sjögren syndrome
★★☆☆2025→ Residue 317
NM_022464.5(SIL1):c.331C>T (p.Arg111Ter)Pathogenic
Marinesco-Sjögren syndrome
★★☆☆2024→ Residue 111
NM_022464.5(SIL1):c.212dup (p.His71fs)Pathogenic
not provided|Marinesco-Sjögren syndrome
★★☆☆2023→ Residue 71
NM_022464.5(SIL1):c.1312C>T (p.Gln438Ter)Pathogenic
Marinesco-Sjögren syndrome|not provided
★★☆☆2021→ Residue 438
NM_022464.5(SIL1):c.178G>T (p.Glu60Ter)Likely pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2025→ Residue 60
NM_022464.5(SIL1):c.453+5G>ALikely pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2024
NM_022464.5(SIL1):c.772_773insGACC (p.Pro258fs)Pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2024→ Residue 258
NM_022464.5(SIL1):c.506_509dup (p.Asp170fs)Pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2024→ Residue 170
NM_022464.5(SIL1):c.1127_1128del (p.Gln376fs)Pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2024→ Residue 376
NM_022464.5(SIL1):c.1029+1G>APathogenic
Marinesco-Sjögren syndrome
★☆☆☆2024
NM_022464.5(SIL1):c.88del (p.Leu30fs)Pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2023→ Residue 30
NM_022464.5(SIL1):c.685dup (p.Gln229fs)Pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2023→ Residue 229
NM_022464.5(SIL1):c.1038del (p.Glu347fs)Likely pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2023→ Residue 347
NM_022464.5(SIL1):c.244+1G>APathogenic
not provided
★☆☆☆2022
NM_022464.5(SIL1):c.643_645+21delLikely pathogenic
Marinesco-Sjögren syndrome
★☆☆☆2022
View on ClinVar ↗
Related Genes
MRPS6Protein interaction100%MRPS9Protein interaction100%MRPL36Protein interaction100%MRPS16Protein interaction100%MRPS12Protein interaction100%MRPS5Protein interaction100%
Tissue Expression6 tissues
Liver
100%
Lung
46%
Heart
44%
Ovary
33%
Bone Marrow
30%
Brain
20%
Gene Interaction Network
Click a node to explore
SIL1MRPS6MRPS9MRPL36MRPS16MRPS12MRPS5
PROTEIN STRUCTURE
Preparing viewer…
PDB6LEY · 2.39 Å · X-ray
View on RCSB ↗
Constraintⓘ
LOEUFⓘ
1.02LoF Tolerant
pLIⓘ
0.00Tolerant
Observed/Expected LoF0.79 [0.61–1.02]
RankingsWhere SIL1 stands among ~20K protein-coding genes
  • #6,205of 20,598
    Most Researched77
  • #1,676of 5,498
    Most Pathogenic Variants35
  • #10,071of 17,882
    Most Constrained (LOEUF)1.02
Genes detectedSIL1
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
A 20-year Clinical and Genetic Neuromuscular Cohort Analysis in Lebanon: An International Effort.
PMID: 34602496
J Neuromuscul Dis · 2022
1.00
2
Role of the HSP70 Co-Chaperone SIL1 in Health and Disease.
PMID: 33557244
Int J Mol Sci · 2021
0.90
3
Ceramic laminate veneers: effect of preparation design and ceramic thickness on fracture resistance and marginal quality in vitro.
PMID: 31900673
Clin Oral Investig · 2020
0.80
4
BiP and its nucleotide exchange factors Grp170 and Sil1: mechanisms of action and biological functions.
PMID: 25698114
J Mol Biol · 2015
0.70
5
SIL1 deficiency causes degenerative changes of peripheral nerves and neuromuscular junctions in fish, mice and human.
PMID: 30468864
Neurobiol Dis · 2019
0.60