PNKP

Chr 19AR

polynucleotide kinase 3'-phosphatase

Also known as: AOA4, CMT2B2, EIEE10, MCSZ, PNK

The protein catalyzes 5' phosphorylation and 3' dephosphorylation of nucleic acids in response to ionizing radiation or oxidative damage as part of DNA repair. Autosomal recessive mutations cause microcephaly, seizures, and developmental delay, ataxia-oculomotor apraxia 4, and possibly Charcot-Marie-Tooth disease type 2B2. Pathogenicity results from impaired DNA repair mechanisms following cellular damage.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismARLOEUF 1.213 OMIM phenotypes
Clinical SummaryPNKP
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Gene-Disease Validity (ClinGen)
microcephaly, seizures, and developmental delay · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
📋
ClinVar Variants
81 unique Pathogenic / Likely Pathogenic· 175 VUS of 590 total submissions
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GeneReview available — PNKP
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
1.21LOEUF
pLI 0.000
Z-score 0.70
OE 0.85 (0.611.21)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint
-1.31Z-score
OE missense 1.21 (1.111.31)
383 obs / 317.4 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.85 (0.611.21)
00.351.4
Missense OE1.21 (1.111.31)
00.61.4
Synonymous OE1.49
01.21.6
LoF obs/exp: 23 / 26.9Missense obs/exp: 383 / 317.4Syn Z: -4.53

ClinVar Variant Classifications

590 submitted variants in ClinVar

Classification Summary

Pathogenic44
Likely Pathogenic37
VUS175
Likely Benign259
Benign14
Conflicting45
44
Pathogenic
37
Likely Pathogenic
175
VUS
259
Likely Benign
14
Benign
45
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
34
3
7
0
44
Likely Pathogenic
29
4
3
1
37
VUS
4
146
20
5
175
Likely Benign
0
6
128
125
259
Benign
0
0
12
2
14
Conflicting
45
Total67159170133574

LoF = frameshift, stop gained/lost, canonical splice · Counts from ClinVar esearch · Updated hourly

View in ClinVar →

Protein Context — Lollipop Plot

PNKP · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

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Clinical Literature
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