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4H syndrome

Genetic disease caused by damage to RNA polymerase III that affects neuronal myelination.

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Concept

4H syndrome

Genetic disease caused by damage to RNA polymerase III that affects neuronal myelination.

It is a rare genetic disease, whose name refers to a set of clinical features that include hypomyelination.

It arises from mutations in subunits of RNA polymerase III, the enzyme responsible for transcribing small RNAs essential for nervous system development, illustrating the consequences of a failure in transcription on brain development.

The video explains that the damage occurs in subunits of RNA polymerase III and that, without that machinery functioning correctly, neuronal myelination fails from early stages, affecting movement, coordination, teeth, and puberty.

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Sources · original ES transcript

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