Au-Kline syndrome
Au-Kline syndrome is a multi-system disorder characterized by hypotonia, intellectual disability, developmental delays, and distinctive facial features.
Overview
Au-Kline syndrome is a condition that affects many body systems. Individuals typically present with weak muscle tone (hypotonia), intellectual disability, and delayed development. Speech and motor skill acquisition are often significantly delayed.
Symptoms
The clinical features of Au-Kline syndrome are varied and may include:
- Developmental delays: Including delayed speech and walking.
- Facial features: Long palpebral fissures, drooping eyelids (ptosis), shallow eye sockets, a broad nasal bridge, and a mouth held in an open position.
- Oral abnormalities: A deep groove in the middle of the tongue, high-arched palate, or missing teeth (oligodontia).
- Structural malformations: Heart, blood vessel, kidney, or bone abnormalities, such as aortic dilatation or spinal curvature (scoliosis).
- Autonomic nervous system issues: Digestive problems, difficulty feeling pain, abnormal sweating, and inability to adjust to high heat.
Causes
Au-Kline syndrome is caused by mutations in the HNRNPK gene. This gene produces the hnRNP K protein, which acts as a docking station for DNA, RNA, and other proteins, helping to control gene activity and protein production. A shortage of this protein disrupts the normal development and function of several body systems, particularly the brain, leading to the symptoms associated with the syndrome.
Inheritance
This condition is inherited in an autosomal dominant pattern, meaning one copy of the altered gene in each cell is sufficient to cause the disorder. Most cases result from new (de novo) mutations that occur during the formation of reproductive cells or in early embryonic development, meaning there is typically no family history of the disorder.