PRDM16

Chr 1AD

PR/SET domain 16

Also known as: CMD1LL, KMT8F, LVNC8, MEL1, PFM13

This gene encodes a transcription regulator that acts as both a histone methyltransferase and chromatin adapter, controlling brown adipocyte differentiation, regulatory T-cell development, and cardiac mitochondrial function. Mutations cause dilated cardiomyopathy and left ventricular noncompaction with autosomal dominant inheritance. The gene is highly constrained against loss-of-function mutations (pLI 0.9999, LOEUF 0.187), indicating that pathogenic variants are likely to have severe functional consequences.

GeneReviewsOMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismADLOEUF 0.192 OMIM phenotypes
Clinical SummaryPRDM16
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Gene-Disease Validity (ClinGen)
dilated cardiomyopathy · ADStrong

Strong evidence — appropriate for clinical testing

Population Constraint (gnomAD)
Highly constrained gene — heterozygous loss-of-function variants are very rare in the population (pLI 1.00). One damaged copy is likely sufficient to cause disease.
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ClinVar Variants
34 unique Pathogenic / Likely Pathogenic· 573 VUS of 1000 total submissions
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Clinical Trials
1 active or recruiting trial — potential therapeutic options may be available
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GeneReview available — PRDM16
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.19LOEUF
pLI 1.000
Z-score 5.95
OE 0.08 (0.040.19)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint
1.35Z-score
OE missense 0.87 (0.820.93)
745 obs / 855.9 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.08 (0.040.19)
00.351.4
Missense OE0.87 (0.820.93)
00.61.4
Synonymous OE1.10
01.21.6
LoF obs/exp: 4 / 48.9Missense obs/exp: 745 / 855.9Syn Z: -1.64
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedPRDM16-related dilated cardiomyopathyOTHERAD
DN
0.3395th %ile
GOF
0.2696th %ile
LOF
0.83top 5%

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

LOFprediction above median · 1 literature citation · 24% of P/LP variants are LoF · LOEUF 0.19

Literature Evidence

LOFSimilarly, haploinsufficiency of PRDM16-a gene which was recently shown to be sufficient to cause the left ventricular noncompaction-SKI, PRKCZ, RERE, UBE4B and MASP2 may contribute to the development of cardiomyopathy.PMID:24454898

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

1000 submitted variants in ClinVar

Classification Summary

Pathogenic28
Likely Pathogenic6
VUS573
Likely Benign360
Benign12
Conflicting16
28
Pathogenic
6
Likely Pathogenic
573
VUS
360
Likely Benign
12
Benign
16
Conflicting

Curated Variants Distribution

Classified variants from ClinVar · 5 ACMG categories

ClassificationLoFMissense + InframeNon-codingSynonymousTotal
Pathogenic
3
0
25
0
28
Likely Pathogenic
5
0
1
0
6
VUS
30
491
44
8
573
Likely Benign
0
4
118
238
360
Benign
0
0
11
1
12
Conflicting
16
Total38495199247995

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

View in ClinVar →

Protein Context — Lollipop Plot

PRDM16 · protein map & ClinVar variants

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

3D Protein StructureAlphaFold
Clinical Literature
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