ACTA1

Chr 1

actin alpha 1, skeletal muscle

Also known as: ACTA, ASMA, CFTD, CFTD1, CFTDM, CMYO2A, CMYO2B, CMYO2C

The product encoded by this gene belongs to the actin family of proteins, which are highly conserved proteins that play a role in cell motility, structure and integrity. Alpha, beta and gamma actin isoforms have been identified, with alpha actins being a major constituent of the contractile apparatus, while beta and gamma actins are involved in the regulation of cell motility. This actin is an alpha actin that is found in skeletal muscle. Mutations in this gene cause a variety of myopathies, including nemaline myopathy, congenital myopathy with excess of thin myofilaments, congenital myopathy with cores, and congenital myopathy with fiber-type disproportion, diseases that lead to muscle fiber defects with manifestations such as hypotonia. [provided by RefSeq, Sep 2019]

ResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProtCongenital myopathy 2A, typical, autosomal dominant
UniProtCongenital myopathy 2B, severe infantile, autosomal recessive
UniProtCongenital myopathy 2C, severe infantile, autosomal dominant
UniProtMyopathy, scapulohumeroperoneal

Clinical highlights

Gene-disease validity (ClinGen)
alpha-actinopathy · ARDefinitivesufficient evidence for diagnostic panels2 gene-disease associations curated in total
0
Active trials
47
Pubs (1 yr)
P/LP submissions
P/LP missense
1.22
LOEUF
DN
Mechanism· predicted
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Missense constrained — critical functional residues
LoF Constraint?
1.22LOEUF
pLI 0.000
Z-score 0.93
OE 0.74 (0.461.22)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint?
4.53Z-score
OE missense 0.21 (0.170.26)
55 obs / 260.9 exp
Constrained

Highly missense-constrained (top ~0.1%)

Observed / Expected Ratios?
LoF OE?0.74 (0.461.22)
00.351.4
Missense OE?0.21 (0.170.26)
00.61.4
Synonymous OE?0.87
01.21.6
LoF obs/exp: 11 / 14.9Missense obs/exp: 55 / 260.9Syn Z: 1.11

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

ACTA1 · protein map & ClinVar variants

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

VCEP specificationsCongenital MyopathiesReleased
Panel ↗

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

No active trials found for this gene.

Search ClinicalTrials.gov →