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Eye Movement Disorders with CACNA1A Mutations

OMIM ID:

autosomal dominant

Eye Movement Disorders with CACNA1A Mutations

Defective Genes

CACNA1A

Clinical Characteristics

Ocular Features

Eye movement disorders secondary to CACNA1A mutations include congenital nystagmus, abnormal saccades and paroxysmal tonic upgaze and can be early indicators of underlying neurologic disease.  The median age of presentation in one series was 1.2 years.

Systemic Features

Eye movement disorders form a group of conditions that may occur in isolation but can also be associated with underlying neurological disease (vida infra).

Genetics

Inheritance

Heterozygous mutations in the CACNA1A gene (19p13.13) have been associated with a number of conditions including type 2 episodic ataxia (108500), familial hemiplegic migraine 1 (141500), and 2 (602481), spinocerebellar ataxia 6 (183086), and several types of eye movement disorders including congenital nystagmus, abnormal saccades, and paroxysmal tonic upgaze. 

The gene product is a transmembrane pore-forming subunit of a voltage-gated calcium channel expressed abundantly in neuronal tissue.

Pedigree

Autosomal dominant

Autosomal dominant disorders require only one mutation for the disease to be expressed.  Since an affected parent has two chromosomes, only one of which has the mutant gene, parents can expect that half (50%) of their children will receive that one and inherit the disease.  It is common for individuals that inherit the mutation, however, to not have evidence of the disease (nonpenetrance).

Image
Sample pedigree of autosomal dominant inheritance

Autosomal dominant inheritance leads to a vertical pattern of transmission

Treatment & Management

The use of calcium channel blockers may have some benefit in preventing severe hemiplegic migraine.

Selected Resources

Web Resources

Publications

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Eye movement disorders are an early manifestation of CACNA1A mutations in children

PubMedID: 26814174