HomeAboutRankingsData Sources
Β© 2026 GeneE
🧬
GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 15 days ago
β“˜GeneE is for informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment.
PKHD1L1
PKHD1 like 1
Chromosome 8 Β· 8q23.1-q23.2
NCBI Gene: 93035Ensembl: ENSG00000205038.12HGNC: HGNC:20313UniProt: Q86WI1
15PubMed Papers
21Diseases
0Drugs
7Pathogenic Variants
FUNCTIONAL ROLE
Receptor
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Swiss-Prot Reviewed
signaling receptor activityimmune responseGO:0005615cytosolautosomal recessive nonsyndromic hearing loss 124female infertilitytooth diseasepreeclampsia
✦AI Summary

PKHD1L1 encodes a large type I transmembrane protein that serves as a critical component of the stereocilia coat in cochlear hair cells, playing an essential role in auditory function 1. The protein is required for maintaining stereocilia bundle cohesion and structural integrity throughout life, with PKHD1L1-deficient mice showing progressive deterioration of hair bundles starting at 6 weeks of age 2. Mechanistically, PKHD1L1 establishes robust sensory hair bundles during development that are necessary for maintaining bundle function in response to acoustic trauma and aging 2. Loss-of-function mutations destabilize protein structure and folding, compromising stereocilia coat formation 1. PKHD1L1 deficiency causes autosomal recessive nonsyndromic sensorineural hearing loss (DFNB124) in humans, with patients exhibiting mild-moderate to severe hearing impairment 1. Animal models demonstrate progressive high-frequency hearing loss that extends to lower frequencies with age, and increased susceptibility to permanent noise-induced hearing damage 2. The gene's function in hearing is evolutionarily conserved, as zebrafish pkhd1l1 mutations also cause hearing deficits 3. Beyond auditory function, PKHD1L1 shows associations with immune cell infiltration and may have roles in cancer prognosis, though these functions require further investigation 4.

Sources cited
1
PKHD1L1 is a component of stereocilia coat and causes autosomal recessive nonsyndromic hearing loss in humans
PMID: 38459354
2
PKHD1L1 is required for stereocilia bundle maintenance and protection against noise-induced hearing loss
PMID: 39482437
3
PKHD1L1 function in hearing is evolutionarily conserved in zebrafish
PMID: 36960824
4
PKHD1L1 expression correlates with immune cell infiltration in cancer contexts
PMID: 38203530
⚠Limited data available β€” This gene has 4 indexed publications. Summary and analysis may be incomplete.
Disease Associationsβ“˜21
autosomal recessive nonsyndromic hearing loss 124Open Targets
0.59Moderate
female infertilityOpen Targets
0.47Moderate
tooth diseaseOpen Targets
0.26Weak
preeclampsiaOpen Targets
0.25Weak
CholecystitisOpen Targets
0.18Weak
lung adenocarcinomaOpen Targets
0.10Weak
Blackfan-Diamond anemiaOpen Targets
0.08Suggestive
cancerOpen Targets
0.07Suggestive
papillary thyroid carcinomaOpen Targets
0.07Suggestive
intelligenceOpen Targets
0.06Suggestive
X-linked severe congenital neutropeniaOpen Targets
0.06Suggestive
neutropenia, severe congenital, 2, autosomal dominantOpen Targets
0.06Suggestive
nonimmune chronic idiopathic neutropenia of adultsOpen Targets
0.05Suggestive
neutropenia, severe congenital, 10, autosomal recessiveOpen Targets
0.05Suggestive
neoplasmOpen Targets
0.05Suggestive
alpha thalassemia-intellectual disability syndrome type 1Open Targets
0.05Suggestive
Alpha-thalassemia - intellectual disability syndrome linked to chromosome 16Open Targets
0.05Suggestive
sebaceous of Jadassohn nevusOpen Targets
0.05Suggestive
myeloperoxidase deficiencyOpen Targets
0.05Suggestive
X-linked sideroblastic anemia 1Open Targets
0.05Suggestive
Deafness, autosomal recessive, 124UniProt
Pathogenic Variants7
NM_177531.6(PKHD1L1):c.6563del (p.Gly2188fs)Likely pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜…β˜†β˜†β˜†2025β†’ Residue 2188
NM_177531.6(PKHD1L1):c.847C>T (p.Arg283Ter)Likely pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜…β˜†β˜†β˜†2025β†’ Residue 283
NM_177531.6(PKHD1L1):c.10141C>T (p.Arg3381Ter)Pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜†β˜†β˜†β˜†2024β†’ Residue 3381
NM_177531.6(PKHD1L1):c.1813G>A (p.Gly605Arg)Pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜†β˜†β˜†β˜†2024β†’ Residue 605
NM_177531.6(PKHD1L1):c.8452_8468del (p.Leu2818fs)Pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜†β˜†β˜†β˜†2024β†’ Residue 2818
NM_177531.6(PKHD1L1):c.3941G>T (p.Gly1314Val)Pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜†β˜†β˜†β˜†2024β†’ Residue 1314
NM_177531.6(PKHD1L1):c.385G>A (p.Gly129Ser)Pathogenic
Autosomal recessive nonsyndromic hearing loss 124
β˜†β˜†β˜†β˜†2024β†’ Residue 129
View on ClinVar β†—
Related Genes
FCGRTShared pathway100%PRG2Shared pathway100%RFX1Shared pathway100%SP2Shared pathway100%VPREB1Shared pathway100%CLEC10AShared pathway100%
Tissue Expression6 tissues
Lung
100%
Heart
42%
Ovary
41%
Bone Marrow
20%
Brain
10%
Liver
9%
Gene Interaction Network
Click a node to explore
PKHD1L1FCGRTPRG2RFX1SP2VPREB1CLEC10A
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted Β· UniProt Q86WI1
View on AlphaFold β†—
Constraintβ“˜
LOEUFβ“˜
1.05LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.96 [0.87–1.05]
RankingsWhere PKHD1L1 stands among ~20K protein-coding genes
  • #15,658of 20,598
    Most Researched15
  • #3,274of 5,498
    Most Pathogenic Variants7
  • #10,574of 17,882
    Most Constrained (LOEUF)1.05
Genes detectedPKHD1L1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
PKHD1L1, a gene involved in the stereocilia coat, causes autosomal recessive nonsyndromic hearing loss.
PMID: 38459354
Hum Genet Β· 2024
1.00
2
PKHD1L1 is required for stereocilia bundle maintenance, durable hearing function and resilience to noise exposure.
PMID: 39482437
Commun Biol Β· 2024
0.90
3
Systematic Multiomic Analysis of
PMID: 38203530
Int J Mol Sci Β· 2023
0.80
4
Integrative analysis of single-cell and transcriptome sequencing with experimental validation reveals PKHD1L1 as a novel biomarker in lung adenocarcinoma.
PMID: 39843484
Sci Rep Β· 2025
0.70
5
PKHD1L1 is required for stereocilia bundle maintenance, durable hearing function and resilience to noise exposure.
PMID: 38496629
bioRxiv Β· 2024
0.60