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GeneE
10 sources retrieved Β· Most recent: April 2026 Β· Index updated 14 days ago
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AHI1
Abelson helper integration site 1
Chromosome 6 Β· 6q23.3
NCBI Gene: 54806Ensembl: ENSG00000135541.22HGNC: HGNC:21575UniProt: A0A7P0T7Z8
89PubMed Papers
21Diseases
0Drugs
271Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
βœ“ Experimental GO Evidenceβœ“ Swiss-Prot Reviewed
protein bindingciliary basal bodycentrosomecilium assemblyJoubert syndrome 3Joubert syndromeJoubert syndrome with ocular defectretinitis pigmentosa
✦AI Summary

AHI1 (Abelson helper integration site 1) is a cilium-localized protein essential for ciliogenesis and primary cilium formation. It mediates vesicle trafficking and recruits RAB8A to the ciliary basal body, functioning as a component of the tectonic-like complex at the ciliary transition zone to regulate transmembrane protein diffusion 1. AHI1 also promotes classical Wnt signaling and neuronal differentiation, potentially influencing cerebellar development. Clinically, AHI1 mutations cause Joubert syndrome (JS), a genetically heterogeneous primary ciliopathy characterized by distinctive midbrain-hindbrain malformation (molar tooth sign) and variable multi-systemic involvement 23. AHI1 accounts for 8-11% of JS cases, particularly in Arab populations 1. Individuals with AHI1 pathogenic variants require close surveillance for retinal dystrophy, including Leber's congenital amaurosis and retinitis pigmentosa 24. Additional organ involvement may include renal dysfunction and neurological complications such as hypotonia, ataxia, developmental delay, and abnormal eye movements 1. As AHI1 is a frequently mutated gene in ciliopathies affecting cilia-dependent signaling, understanding its transcriptional regulation and functional mechanisms remains critical for developing targeted treatments for ciliary-associated kidney and retinal diseases 5.

Sources cited
1
AHI1 encodes a cilium-localized protein with roles in vesicle trafficking, ciliogenesis, and cell polarity; accounts for 8-11% of JS cases particularly in Arab populations
PMID: 34191236
2
AHI1 variants in JS patients require closer surveillance for retinal dystrophy
PMID: 35238134
3
AHI1 is among causative genes in Joubert syndrome with multi-systemic organ involvement including eye complications
PMID: 36580738
4
AHI1 identified as novel candidate gene for nonsyndromic retinitis pigmentosa in inherited retinal dystrophy cohort
PMID: 25356976
5
Ciliary-associated protein mutations including those affecting ciliogenesis cause renal ciliopathies characterized by kidney dysfunction
PMID: 33039432
Disease Associationsβ“˜21
Joubert syndrome 3Open Targets
0.78Strong
Joubert syndromeOpen Targets
0.76Strong
Joubert syndrome with ocular defectOpen Targets
0.74Strong
retinitis pigmentosaOpen Targets
0.60Moderate
Joubert syndrome and related disordersOpen Targets
0.55Moderate
Retinal dystrophyOpen Targets
0.53Moderate
asthmaOpen Targets
0.50Moderate
Joubert syndrome 1Open Targets
0.48Moderate
Rod-cone dystrophyOpen Targets
0.47Moderate
respiratory system diseaseOpen Targets
0.45Moderate
multiple sclerosisOpen Targets
0.45Moderate
nephronophthisisOpen Targets
0.43Moderate
Leber congenital amaurosisOpen Targets
0.43Moderate
hypothyroidismOpen Targets
0.42Moderate
genetic disorderOpen Targets
0.42Moderate
allergic rhinitisOpen Targets
0.40Weak
preeclampsiaOpen Targets
0.39Weak
atopic eczemaOpen Targets
0.37Weak
eye diseaseOpen Targets
0.37Weak
Joubert syndrome 17Open Targets
0.37Weak
Joubert syndrome 3UniProt
Pathogenic Variants271
NM_001134831.2(AHI1):c.2246C>G (p.Ser749Ter)Pathogenic
Nephronophthisis|Joubert syndrome|Retinal disorder
β˜…β˜…β˜†β˜†2026β†’ Residue 749
NM_001134831.2(AHI1):c.72_85del (p.Ser24fs)Pathogenic
not provided|Joubert syndrome|Joubert syndrome 3|Retinal dystrophy
β˜…β˜…β˜†β˜†2026β†’ Residue 24
NM_001134831.2(AHI1):c.3235C>T (p.Arg1079Ter)Pathogenic
Rod-cone dystrophy|Joubert syndrome 3|Joubert syndrome|not provided
β˜…β˜…β˜†β˜†2026β†’ Residue 1079
NM_001134831.2(AHI1):c.910dup (p.Thr304fs)Pathogenic
Joubert syndrome 3|Joubert syndrome|not provided|Joubert syndrome and related disorders|Retinal dystrophy
β˜…β˜…β˜†β˜†2026β†’ Residue 304
NM_001134831.2(AHI1):c.2105C>T (p.Thr702Met)Pathogenic
Joubert syndrome|Joubert syndrome and related disorders|not provided|Joubert syndrome 3
β˜…β˜…β˜†β˜†2026β†’ Residue 702
NM_001134831.2(AHI1):c.313C>T (p.Gln105Ter)Pathogenic
Joubert syndrome 3|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 105
NM_001134831.2(AHI1):c.108dup (p.Leu37fs)Pathogenic
Joubert syndrome 3|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 37
NM_001134831.2(AHI1):c.3245del (p.Phe1082fs)Pathogenic
Joubert syndrome|Joubert syndrome 3
β˜…β˜…β˜†β˜†2025β†’ Residue 1082
NM_001134831.2(AHI1):c.2174G>A (p.Trp725Ter)Pathogenic
Joubert syndrome 3|Retinitis pigmentosa|Leber congenital amaurosis|not provided|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 725
NM_001134831.2(AHI1):c.96dup (p.Leu33fs)Pathogenic
not provided|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 33
NM_001134831.2(AHI1):c.484C>T (p.Gln162Ter)Pathogenic
Joubert syndrome|Joubert syndrome 3|Joubert syndrome and related disorders|Retinal dystrophy
β˜…β˜…β˜†β˜†2025β†’ Residue 162
NM_001134831.2(AHI1):c.2212C>T (p.Arg738Ter)Pathogenic
Joubert syndrome 3|not provided|Retinitis pigmentosa|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 738
NM_001134831.2(AHI1):c.533_534del (p.Glu178fs)Pathogenic
Joubert syndrome|Joubert syndrome 3
β˜…β˜…β˜†β˜†2025β†’ Residue 178
NM_001134831.2(AHI1):c.1811C>G (p.Ser604Ter)Pathogenic
Retinal dystrophy|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 604
NM_001134831.2(AHI1):c.1267C>T (p.Gln423Ter)Pathogenic
Joubert syndrome 3|Joubert syndrome|not provided|Retinal dystrophy|Nephronophthisis|Rod-cone dystrophy|Joubert syndrome and related disorders
β˜…β˜…β˜†β˜†2025β†’ Residue 423
NM_001134831.2(AHI1):c.3032C>G (p.Ser1011Ter)Pathogenic
not provided|Retinitis pigmentosa|Retinal dystrophy|Joubert syndrome|Joubert syndrome 3|Joubert syndrome and related disorders
β˜…β˜…β˜†β˜†2025β†’ Residue 1011
NM_001134831.2(AHI1):c.2098_2099dup (p.Tyr701fs)Pathogenic
Joubert syndrome 3|not provided|Joubert syndrome
β˜…β˜…β˜†β˜†2025β†’ Residue 701
NM_001134831.2(AHI1):c.2247dup (p.Leu750fs)Pathogenic
Joubert syndrome|not provided|Joubert syndrome 3
β˜…β˜…β˜†β˜†2025β†’ Residue 750
NM_001134831.2(AHI1):c.1166C>G (p.Ser389Ter)Pathogenic
Joubert syndrome|Joubert syndrome 3
β˜…β˜…β˜†β˜†2025β†’ Residue 389
NM_001134831.2(AHI1):c.1626+1G>APathogenic
Joubert syndrome 3|Joubert syndrome|not provided
β˜…β˜…β˜†β˜†2025
View on ClinVar β†—
Related Genes
CC2D2AProtein interaction100%TMEM231Protein interaction100%NPHP3Protein interaction98%TCTN3Protein interaction97%RAB8AProtein interaction96%TMEM107Protein interaction94%
Tissue Expression6 tissues
Bone Marrow
100%
Ovary
89%
Brain
73%
Liver
59%
Heart
51%
Lung
48%
Gene Interaction Network
Click a node to explore
AHI1CC2D2ATMEM231NPHP3TCTN3RAB8ATMEM107
PROTEIN STRUCTURE
Preparing viewer…
PDB4ESR Β· 1.53 Γ… Β· X-ray
View on RCSB β†—
Constraintβ“˜
LOEUFβ“˜
1.07LoF Tolerant
pLIβ“˜
0.00Tolerant
Observed/Expected LoF0.90 [0.77–1.07]
RankingsWhere AHI1 stands among ~20K protein-coding genes
  • #5,347of 20,598
    Most Researched89
  • #232of 5,498
    Most Pathogenic Variants271 Β· top 5%
  • #10,785of 17,882
    Most Constrained (LOEUF)1.07
Genes detectedAHI1
Sources retrieved10 papers
Response timeβ€”
πŸ“„ Sources
10β–Ό
1
Ciliopathies and the Kidney: A Review.
PMID: 33039432
Am J Kidney Dis Β· 2021
1.00
2
Genotype-phenotype correlates in Joubert syndrome: A review.
PMID: 35238134
Am J Med Genet C Semin Med Genet Β· 2022
0.90
3
PMID: 20301500
0.80
4
Genotype-phenotype correlation and mutation spectrum in a large cohort of patients with inherited retinal dystrophy revealed by next-generation sequencing.
PMID: 25356976
Genet Med Β· 2015
0.70
5
Innate depression.
PMID: 35998233
Sci Signal Β· 2022
0.60