Polycystic liver disease 3 with or without kidney cysts

disease
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Also known as PCLD3

Summary

Polycystic liver disease 3 with or without kidney cysts (MONDO:0054743) is a disease caused by ALG8 (GenCC Strong), with 1 cohort gene.

At a glance

  • Causal gene: ALG8 (GenCC Strong)
  • Cohort genes: 1
  • ClinVar variants: 123

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical namepolycystic liver disease 3 with or without kidney cysts
Mondo IDMONDO:0054743
OMIM617874
DOIDDOID:0060976
UMLSC4693472
MedGen1646969
GARD0025967
Is cancer (heuristic)no

Also known as: PCLD3 · polycystic liver disease 3 with or without kidney cysts

Data availability: 123 ClinVar variants · 4 GenCC gene-disease records.

Disease family

Classification path: disease › human disease › disease by etiologic mechanism › disease of genetic or genomic mechanism › hereditary disease › autosomal genetic disease › autosomal dominant disease › autosomal dominant polycystic liver diseasepolycystic liver disease 3 with or without kidney cysts

Related subtypes (4): polycystic liver disease 1, polycystic liver disease 2, polycystic liver disease 4 with or without kidney cysts, SEC61B-related polycystic liver disease

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

123 retrieved; paginated sample, class counts are floors:

72 uncertain significance, 24 likely pathogenic, 10 pathogenic/likely pathogenic, 6 conflicting classifications of pathogenicity, 6 likely benign, 2 benign/likely benign, 2 pathogenic, 1 no classifications from unflagged records

ClinVarVariant (HGVS)GeneClassificationReview
1437904NM_024079.5(ALG8):c.802del (p.Arg268fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2230523NM_024079.5(ALG8):c.309dup (p.Leu104fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2506353NM_024079.5(ALG8):c.272del (p.Asn91fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2561NM_024079.5(ALG8):c.139A>C (p.Thr47Pro)ALG8Pathogeniccriteria provided, single submitter
280116NM_024079.5(ALG8):c.1090C>T (p.Arg364Ter)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
2982294NM_024079.5(ALG8):c.824del (p.Gly275fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
3574056NM_024079.5(ALG8):c.740T>G (p.Leu247Ter)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
4531361NM_024079.5(ALG8):c.967C>T (p.Gln323Ter)ALG8Pathogeniccriteria provided, single submitter
492977NM_024079.5(ALG8):c.535C>T (p.Arg179Ter)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
854540NM_024079.5(ALG8):c.761dup (p.Pro255fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
956334NM_024079.5(ALG8):c.981dup (p.Val328fs)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
96090NM_024079.5(ALG8):c.121C>T (p.Arg41Ter)ALG8Pathogenic/Likely pathogeniccriteria provided, multiple submitters, no conflicts
1328207NM_024079.5(ALG8):c.685C>T (p.Arg229Ter)ALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
1333512NM_024079.5(ALG8):c.710_719dup (p.Leu241fs)ALG8Likely pathogeniccriteria provided, single submitter
1344961NM_024079.5(ALG8):c.95+1G>AALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
2562NM_024079.5(ALG8):c.673+4A>GALG8Likely pathogeniccriteria provided, single submitter
2563NM_024079.5(ALG8):c.824G>A (p.Gly275Asp)ALG8Likely pathogeniccriteria provided, single submitter
2683953NM_024079.5(ALG8):c.544C>T (p.Gln182Ter)ALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
3574028NM_024079.5(ALG8):c.1354G>T (p.Glu452Ter)ALG8Likely pathogeniccriteria provided, single submitter
3574032NM_024079.5(ALG8):c.1276+1G>AALG8Likely pathogeniccriteria provided, single submitter
3574034NM_024079.5(ALG8):c.1218del (p.Phe406_Leu407insTer)ALG8Likely pathogeniccriteria provided, single submitter
3574035NM_024079.5(ALG8):c.1184_1187delinsGTT (p.Leu395fs)ALG8Likely pathogeniccriteria provided, single submitter
3574036NM_024079.5(ALG8):c.1179-7_1179-2delALG8Likely pathogeniccriteria provided, single submitter
3574038NM_024079.5(ALG8):c.1131del (p.Trp378fs)ALG8Likely pathogeniccriteria provided, single submitter
3574039NM_024079.5(ALG8):c.1114dup (p.Ser372fs)ALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
3574041NM_024079.5(ALG8):c.1057del (p.Trp353fs)ALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
3574046NM_024079.5(ALG8):c.867C>G (p.Tyr289Ter)ALG8Likely pathogeniccriteria provided, single submitter
3574061NM_024079.5(ALG8):c.674-2A>GALG8Likely pathogeniccriteria provided, multiple submitters, no conflicts
3574064NM_024079.5(ALG8):c.643C>T (p.Arg215Ter)ALG8Likely pathogeniccriteria provided, single submitter
3574073NM_024079.5(ALG8):c.219G>A (p.Trp73Ter)ALG8Likely pathogeniccriteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 9 · Orphanet: 2 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

GenCC gene–disease validity (cohort genes)

the Disease column is the GenCC-asserted condition — a cohort gene’s strongest validity may be for a related predisposition syndrome.

GeneClassificationInheritanceDiseaseRecords
ALG8StrongAutosomal dominantpolycystic liver disease 3 with or without kidney cysts9

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
ALG8Orphanet:2924Isolated polycystic liver disease
ALG8Orphanet:79325ALG8-CDG

Cohort genes → proteins

1 cohort genes, 1 distinct canonical proteins.

Evidence partition

SubsetGenes
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
ALG8HGNC:23161ENSG00000159063Q9BVK2Dolichyl pyrophosphate Glc1Man9GlcNAc2 alpha-1,3-glucosyltransferasegencc,clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
ALG8Dolichyl pyrophosphate Glc1Man9GlcNAc2 alpha-1,3-glucosyltransferaseDolichyl pyrophosphate Glc1Man9GlcNAc2 alpha-1,3-glucosyltransferase that operates in the biosynthetic pathway of dolichol-linked oligosaccharides, the glycan precursors employed in protein asparagine (N)-glycosylation.

Protein-family classification

Druggable: 1 · Difficult: 0 · Unknown: 0 · Druggable fraction: 1.0

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

FamilyGenesFoldFDR
Enzyme (other)112.0×0.083

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
ALG8Enzyme (other)yes2.4.1.265Glyco_trans_ALG6/ALG8

Expression context

Cohort genes with no expression data: 0.

1 cohort gene are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)1
unknown0

Top tissues across cohort

TissueCohort genes
left testis1
right testis1
testis1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
ALG8282ubiquitousmarkerright testis, left testis, testis

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
ALG81,010

Structural data

PDB: 0 · AlphaFold-only: 1 · No structure: 0

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
ALG8Q9BVK292.07

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 9. Enrichment computed across 1 evidence-associated genes (1 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
Defective ALG8 causes CDG-1h111420.0×8e-04ALG8
Diseases associated with N-glycosylation of proteins1634.4×0.007ALG8
Biosynthesis of the N-glycan precursor (dolichol lipid-linked oligosaccharide, LLO) and transfer to a nascent protein1207.6×0.014ALG8
Diseases of glycosylation1131.3×0.017ALG8
Diseases of metabolism180.4×0.022ALG8
Asparagine N-linked glycosylation160.1×0.025ALG8
Post-translational protein modification119.2×0.067ALG8
Disease113.1×0.081ALG8
Metabolism of proteins112.4×0.081ALG8

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 1 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
dolichol-linked oligosaccharide biosynthetic process1842.6×0.002ALG8
obsolete protein N-linked glycosylation via asparagine1674.1×0.002ALG8
protein N-linked glycosylation1263.3×0.004ALG8

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 1

Druggability breadth: 1 of 1 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

SymbolMoleculesMax phase
ALG800

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

SymbolAssaysType breakdown
ALG81Binding:1

Cohort enzymes (BRENDA EC)

SymbolEC numbersNames
ALG82.4.1.265dolichyl-P-Glc:Glc1Man9GlcNAc2-PP-dolichol alpha-1,3-glucosyltransferase

Pharmacogenomics

Cohort genes with a PharmGKB record: 1; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

TierDefinitionGenesSymbols
AApproved (phase 4 drug)0
BPhased (≥1) drug, not yet approved0
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug1ALG8
EDifficult family or no structure, no drug0

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

SymbolChEMBL assaysDrugged partners (top 3)
ALG81

Clinical trials & evidence

Clinical trials

Clinical trials: 0.