PTHLH

Chr 12AD

parathyroid hormone like hormone

Also known as: BDE2, HHM, PLP, PTHR, PTHRP

The protein encoded by this gene is a neuroendocrine peptide that regulates cellular growth, development, and survival, and specifically controls endochondral bone development and epithelial-mesenchymal interactions during formation of mammary glands and teeth through its receptor PTH1R. Mutations cause brachydactyly type E2, characterized by shortened digits, and follow an autosomal dominant inheritance pattern. This gene is highly constrained against loss-of-function variants, indicating that such mutations are likely to have significant clinical consequences.

Summary from RefSeq, OMIM, UniProt
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Primary Disease Associations & Inheritance

Brachydactyly, type E2MIM #613382
AD
UniProtBrachydactyly E2
0
Active trials
32
Pubs (1 yr)
59
P/LP submissions
13%
P/LP missense
0.48
LOEUF
LOF
Mechanism· G2P
Clinical SummaryPTHLH
Population Constraint (gnomAD)
Moderately constrained gene (pLI 0.81) — some intolerance to loss-of-function variants.
📋
ClinVar Variants
56 unique Pathogenic / Likely Pathogenic· 71 VUS of 165 total submissions
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GeneReview available — PTHLH
Authoritative clinical overview · Recommended first read
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Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.48LOEUF
pLI 0.811
Z-score 2.62
OE 0.10 (0.040.48)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
1.15Z-score
OE missense 0.69 (0.570.83)
74 obs / 107.7 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.10 (0.040.48)
00.351.4
Missense OE0.69 (0.570.83)
00.61.4
Synonymous OE0.74
01.21.6
LoF obs/exp: 1 / 9.9Missense obs/exp: 74 / 107.7Syn Z: 1.39
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitivePTHLH-related brachydactylyLOFAD
definitivePTHLH-related clubbing with skeletal dysplasia with acroosteolysisOTHERAD
DN
0.4190th %ile
GOF
0.4382th %ile
LOF
0.69top 10%

The highest-scoring mechanism for this gene is loss-of-function (haploinsufficiency).

LOFprediction above median · 1 literature citation · LOEUF 0.48

Literature Evidence

LOFHere, we report three patients affected with brachydactyly type E, caused by PTHLH mutations expected to result in haploinsufficiency, and discuss our data compared to published reports.PMID:26640227

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312. Mechanism ranking also informed by gnomAD constraint, ClinVar, and ClinGen data.

ClinVar Variant Classifications

165 submitted variants in ClinVar

Classification Summary

Pathogenic46
Likely Pathogenic10
VUS71
Likely Benign27
Benign4
Conflicting1
46
Pathogenic
10
Likely Pathogenic
71
VUS
27
Likely Benign
4
Benign
1
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
3
40
0
46
Likely Pathogenic
3
4
3
0
10
VUS
2
62
6
1
71
Likely Benign
0
1
6
20
27
Benign
0
0
4
0
4
Conflicting
1
Total8705921159

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

View in ClinVar →

Protein Context — Lollipop Plot

PTHLH · protein map & ClinVar variants

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

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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