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10 sources retrieved · Most recent: April 2026 · Index updated 15 days ago
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PHKA2
phosphorylase kinase regulatory subunit alpha 2
Chromosome X · Xp22.13
NCBI Gene: 5256Ensembl: ENSG00000044446.13HGNC: HGNC:8926UniProt: P46019
78PubMed Papers
21Diseases
0Drugs
118Pathogenic Variants
RESEARCH IMPACT
Variant-Rich
CLINICAL
OMIM Disease Gene
DATA QUALITY
✓ Experimental GO Evidence✓ Swiss-Prot Reviewed
protein bindingphosphorylase kinase complexphosphorylase kinase activitycarbohydrate metabolic processglycogen storage disease IXa1disorder of glycogen metabolismGlycogen storage disease due to glycogenin deficiencygenetic disorder
✦AI Summary

PHKA2 encodes the liver α-subunit of phosphorylase kinase, a key regulatory enzyme in carbohydrate metabolism 1. The protein functions as part of the phosphorylase kinase complex, which catalyzes phosphorylation of glycogen phosphorylase to mobilize hepatic glycogen stores and generate energy 2. The PHKA2 gene encodes a 1235 amino acid polypeptide involved in carbohydrate metabolic processes and protein modification 1. PHKA2 mutations cause glycogen storage disease type IXa (GSD IXa), the most frequent liver glycogen storage disease 12. Clinical presentations include hepatomegaly, elevated liver transaminases, ketotic hypoglycemia, hyperlipidemia, and poor growth, though phenotypic variability exists 23. Missense mutations can produce variant biochemical phenotypes with tissue-specific enzyme activity differences 4. Rare cases exhibit systemic complications including renal tubular dysfunction, neurological symptoms, or progressive cirrhosis 45. Beyond classical metabolism, emerging evidence suggests PHKA2 participates in glycolipid metabolism regulation via post-translational modification of transcriptional regulators, potentially relevant to glioblastoma pathogenesis 6. Genetic testing enables accurate GSD IXa diagnosis and guides management through dietary intervention and supportive therapy 7.

Sources cited
1
PHKA2 encodes a 1235 amino acid liver phosphorylase kinase α-subunit; mutations cause X-linked liver glycogenosis/GSD IXa
PMID: 7549948
2
GSD IX is caused by phosphorylase kinase deficiency; PHKA2 mutations result in hepatomegaly, elevated transaminases, ketotic hypoglycemia, and poor growth
PMID: 30659246
3
PHKA2 mutations cause GSD IXa; hypoglycemia may be an early symptom in infants
PMID: 30925902
4
PHKA2 mutations show phenotypic heterogeneity, including variant biochemical phenotypes and uncommon clinical features involving kidneys and nervous system
PMID: 9600238
5
PHKA2 mutations can rarely cause severe liver cirrhosis in young children with GSD IXa
PMID: 34727590
6
PHKA2 participates in glycolipid metabolism regulation through phosphorylation of transcriptional regulators
PMID: 39794701
7
PHKA2 genetic testing enables GSD IXa diagnosis; high-protein/starch diet and supportive therapy improve outcomes
PMID: 31725618
Disease Associationsⓘ21
glycogen storage disease IXa1Open Targets
0.82Strong
disorder of glycogen metabolismOpen Targets
0.64Moderate
Glycogen storage disease due to glycogenin deficiencyOpen Targets
0.50Moderate
genetic disorderOpen Targets
0.50Moderate
Glycogen storage disease due to phosphorylase kinase deficiencyOpen Targets
0.47Moderate
glycogen storage disease due to liver phosphorylase kinase deficiencyOpen Targets
0.37Weak
Aland island eye diseaseOpen Targets
0.34Weak
Increased hepatic glycogen contentOpen Targets
0.26Weak
Intellectual disabilityOpen Targets
0.12Weak
neoplasmOpen Targets
0.08Suggestive
glioblastoma multiformeOpen Targets
0.07Suggestive
infectionOpen Targets
0.03Suggestive
cancerOpen Targets
0.03Suggestive
hyperinsulinemic hypoglycemia, familial, 4Open Targets
0.02Suggestive
HypoglycemiaOpen Targets
0.02Suggestive
malignant hyperthermia, susceptibility to, 1Open Targets
0.02Suggestive
clear cell renal carcinomaOpen Targets
0.02Suggestive
glycogen storage disease IXOpen Targets
0.02Suggestive
blue cone monochromacyOpen Targets
0.01Suggestive
glioblastomaOpen Targets
0.01Suggestive
Glycogen storage disease 9AUniProt
Pathogenic Variants118
NM_000292.3(PHKA2):c.133C>T (p.Arg45Trp)Pathogenic
Glycogen storage disease IXa1|not provided
★★☆☆2025→ Residue 45
NM_000292.3(PHKA2):c.3614C>T (p.Pro1205Leu)Pathogenic
Glycogen storage disease IXa1|not provided|Glycogen storage disease
★★☆☆2025→ Residue 1205
NM_000292.3(PHKA2):c.1174C>T (p.Arg392Ter)Pathogenic
Glycogen storage disease IXa1
★★☆☆2025→ Residue 392
NM_000292.3(PHKA2):c.4C>G (p.Arg2Gly)Pathogenic
Glycogen storage disease IXa1|not provided|Glycogen phosphorylase kinase deficiency|PHKA2-related disorder|X-linked PHKA2-related disorders
★★☆☆2025→ Residue 2
NM_000292.3(PHKA2):c.2597+1G>APathogenic
Glycogen storage disease IXa1|not provided
★★☆☆2025
NM_000292.3(PHKA2):c.256C>T (p.Arg86Ter)Pathogenic
Glycogen storage disease IXa1|Inborn genetic diseases
★★☆☆2025→ Residue 86
NM_000292.3(PHKA2):c.134G>A (p.Arg45Gln)Pathogenic
Glycogen storage disease IXa1|not provided
★★☆☆2025→ Residue 45
NM_000292.3(PHKA2):c.3629G>A (p.Gly1210Glu)Pathogenic
Glycogen storage disease IXa1
★★☆☆2024→ Residue 1210
NM_000292.3(PHKA2):c.883C>T (p.Arg295Cys)Pathogenic
Glycogen storage disease IXa1|Glycogen phosphorylase kinase deficiency
★★☆☆2024→ Residue 295
NM_000292.3(PHKA2):c.2746C>T (p.Arg916Trp)Pathogenic
not provided|Glycogen storage disease IXa1
★★☆☆2024→ Residue 916
NM_000292.3(PHKA2):c.3331C>T (p.Arg1111Ter)Pathogenic
Glycogen storage disease IXa1
★★☆☆2024→ Residue 1111
NM_000292.3(PHKA2):c.557G>A (p.Arg186His)Likely pathogenic
Glycogen storage disease IXa2|Glycogen storage disease IXa1
★★☆☆2023→ Residue 186
NM_000292.3(PHKA2):c.1205G>A (p.Trp402Ter)Pathogenic
Glycogen storage disease IXa1|not provided
★★☆☆2023→ Residue 402
NM_000292.3(PHKA2):c.1A>G (p.Met1Val)Pathogenic
Glycogen storage disease IXa1|not provided
★★☆☆2023→ Residue 1
NM_000292.3(PHKA2):c.918+1G>APathogenic
Glycogen storage disease IXa1|Increased hepatic glycogen content|not provided
★★☆☆2023
NM_000292.3(PHKA2):c.3210_3212del (p.Arg1072del)Pathogenic
not provided|Glycogen storage disease IXa1
★★☆☆2022→ Residue 1072
NM_000292.3(PHKA2):c.556C>T (p.Arg186Cys)Likely pathogenic
Glycogen storage disease IXa2|Glycogen storage disease IXa1|not provided
★★☆☆2021→ Residue 186
NM_000292.3(PHKA2):c.2909-1G>TLikely pathogenic
Glycogen storage disease IXa1
★☆☆☆2026
NM_000292.3(PHKA2):c.537+2T>CLikely pathogenic
Nonpapillary renal cell carcinoma|Glycogen storage disease IXa1
★☆☆☆2025
NM_000292.3(PHKA2):c.347A>G (p.Tyr116Cys)Likely pathogenic
Glycogen storage disease
★☆☆☆2025→ Residue 116
View on ClinVar ↗
Related Genes
CALM1Protein interaction97%PYGBProtein interaction96%CALM2Protein interaction96%CALM3Protein interaction87%CALML5Protein interaction87%CALML3Protein interaction87%
Tissue Expression6 tissues
Liver
100%
Ovary
45%
Bone Marrow
33%
Heart
28%
Lung
22%
Brain
8%
Gene Interaction Network
Click a node to explore
PHKA2CALM1PYGBCALM2CALM3CALML5CALML3
PROTEIN STRUCTURE
Preparing viewer…
AlphaFoldAI-predicted · UniProt P46019
View on AlphaFold ↗
Constraintⓘ
LOEUFⓘ
0.41Moderately Constrained
pLIⓘ
1.00Intolerant
Observed/Expected LoF0.30 [0.23–0.41]
RankingsWhere PHKA2 stands among ~20K protein-coding genes
  • #6,101of 20,598
    Most Researched78
  • #658of 5,498
    Most Pathogenic Variants118 · top quartile
  • #2,114of 17,882
    Most Constrained (LOEUF)0.41 · top quartile
Genes detectedPHKA2
Sources retrieved10 papers
Response time—
📄 Sources
10▼
1
Diagnosis and management of glycogen storage diseases type VI and IX: a clinical practice resource of the American College of Medical Genetics and Genomics (ACMG).
PMID: 30659246
Genet Med · 2019
1.00
2
Isolation of cDNA encoding the human liver phosphorylase kinase alpha subunit (PHKA2) and identification of a missense mutation of the PHKA2 gene in a family with liver phosphorylase kinase deficiency.
PMID: 7549948
Biochem Mol Biol Int · 1995
0.90
3
Effect of SNORD113-3/ADAR2 on glycolipid metabolism in glioblastoma via A-to-I editing of PHKA2.
PMID: 39794701
Cell Mol Biol Lett · 2025
0.80
4
A novel PHKA2 mutation in a Chinese child with glycogen storage disease type IXa: a case report and literature review.
PMID: 30925902
BMC Med Genet · 2019
0.70
5
A novel
PMID: 38192425
Front Endocrinol (Lausanne) · 2023
0.60