LMNA

Chr 1ADAR

lamin A/C

Also known as: CDCD1, CDDC, CMD1A, CMT2B1, EMD2, FPL, FPLD, FPLD2

The encoded lamin A/C protein forms part of the nuclear lamina matrix that maintains nuclear stability and regulates chromatin structure and gene expression. Mutations cause a diverse spectrum of diseases including Emery-Dreifuss muscular dystrophy, dilated cardiomyopathy, Charcot-Marie-Tooth disease type 2B1, Hutchinson-Gilford progeria syndrome, familial partial lipodystrophy, and mandibuloacral dysplasia, inherited in both autosomal dominant and autosomal recessive patterns. The mechanism of pathogenicity is predicted to be dominant negative, where mutant proteins disrupt normal nuclear lamina function.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
GOF/LOFmechanismAD/ARLOEUF 0.2111 OMIM phenotypes
Clinical SummaryLMNA
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Gene-Disease Validity (ClinGen)
lipodystrophy · SDDefinitive

Definitive — sufficient evidence for diagnostic panels

3 total gene-disease associations curated

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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Clinical Trials
7 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint
0.21LOEUF
pLI 0.999
Z-score 4.75
OE 0.07 (0.030.21)
Highly constrained

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

Missense Constraint
2.37Z-score
OE missense 0.67 (0.600.74)
269 obs / 402.7 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios
LoF OE0.07 (0.030.21)
00.351.4
Missense OE0.67 (0.600.74)
00.61.4
Synonymous OE0.95
01.21.6
LoF obs/exp: 2 / 30.2Missense obs/exp: 269 / 402.7Syn Z: 0.55
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
limitedLMNA-related arrhythmogenic right ventricular cardiomyopathyOTHERAD
definitiveLMNA-related dilated cardiomyopathyOTHERAD
definitiveLMNA-related Hutchinson-Gilford progeria syndromeGOFAD
limitedLMNA-related lethal tight skin contracture syndromeGOFAD
limitedLMNA-related Charcot-Marie-Tooth diseaseOTHERAR
definitiveLMNA-related Emery-Dreifuss muscular dystrophyLOFAD
moderateLMNA-related heart-hand syndrome, Slovenian typeOTHERAD
definitiveLMNA-related familial partial lipodystrophyOTHERAD
definitiveLMNA-related restrictive dermopathy, lethalLOFAR
Mechanism Note (variant-dependent)
DNLOF— mechanism depends on specific variant

Lamin A/C. DN missense variants cause EDMD, dilated cardiomyopathy, lipodystrophy, and progeria. LOF (haploinsufficiency) also causes dilated cardiomyopathy. Biallelic LOF causes restrictive dermopathy. Mechanism is variant- and phenotype-dependent.

DN
0.76top 25%
GOF
0.6638th %ile
LOF
0.54top 25%

This gene has evidence for multiple mechanisms of pathogenicity (loss-of-function, dominant-negative and gain-of-function). The Badonyi & Marsh model scores dominant-negative highest among its predictions, but genomic evidence (constraint, ClinVar variant spectrum, and literature) most strongly supports loss-of-function (haploinsufficiency). Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

LOF1 literature citation · LOEUF 0.21
DNprediction above median · 1 literature citation
GOFprediction above median · 1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNHutchinson-Gilford progeria syndrome (HGPS or progeria) is typically caused by a dominant-negative C:G-to-T:A mutation (c.1824 C>T; p.G608G) in LMNA, the gene that encodes nuclear lamin A.PMID:33408413
GOFThe findings of this study suggested that mutations responsible for FPLD are gain-of-function mutations. Boguslavsky et al. (2006) postulated that mutations that result in gain of function may cause higher binding affinity to a proadipogenic transcription factor, thus preventing it from activating tPMID:16415042
LOFWe hypothesize that the decreased cardiac function and contractures may be related to LMNA haploinsufficiency.PMID:22903861

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

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

LMNA · 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

Lipodystrophy (Genetic or Acquired, Non HIV)

The LD Lync Study - Natural History Study of Lipodystrophy Syndromes

RECRUITING
NCT03087253University of MichiganStarted 2018-02-27
Arrhythmogenic Cardiomyopathies

Tissue and Metabolic Characterization of Arrhythmogenic Cardiomyopathies by Hybrid PET-MRI Imaging, Impact of the Observed Profiles on the Phenotype and on the Evolution of Cardiomyopathy

RECRUITING
NCT05450783Nantes University HospitalStarted 2022-09-01
Biocollection
Polycystic Ovary SyndromeFamilial Partial LipodystrophyLMNA (LaMin Nuclear A) Related Disorders

Identification of Women With Severe Insulin Resistant Syndromes of Genetic Origin Among Patients With "Classic" Polycystic Ovary Syndrome (PCOS)

NOT YET RECRUITING
NCT07412028Phase NAAssistance Publique - Hôpitaux de ParisStarted 2026-02
Genetic analysisBiological analysisimaging test
Lipodystrophy

Assess the Possibility of Diagnosing Diabetes and Rediabetes Following Oral Induced Hyperglycemia in Patients With Dunnigan's Partial Familial Lipodystrophy by Replacing 75 g of Glucose With a Standardized Carbohydrate Breakfast and Continuous Interstitial Monitoring Glucose)

RECRUITING
NCT05789251Phase NACentre Hospitalier Universitaire de la RéunionStarted 2023-06-19
Standardized breakfast
CardiomyopathiesGenetic PredispositionCardiomyopathy, Primary

Biomarkers in SCOTland CardiomyopatHy Registry (Bio-SCOTCH)

RECRUITING
NCT06446271NHS Greater Glasgow and ClydeStarted 2024-06-26
Plasma biomarker levels
LaminopathiesEmerinopathies

Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies)

RECRUITING
NCT03058185Pitié-Salpêtrière HospitalStarted 2013-07-11
LaminopathiesEmery Dreifuss Muscular Dystrophy 2LMNA-Related Congenital Muscular Dystrophy

Modifying Factors in Striated Muscle Laminopathies

RECRUITING
NCT05394506Phase NAInstitut National de la Santé Et de la Recherche Médicale, FranceStarted 2022-09-08
Skin BiopsyMuscle biopsy
Clinical Literature
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