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CHASERR deletion

Epidemilogy: - rare, 3 cases Pathology: - deletions of CHASERR, a long non-coding RNA - results in increased expression of a nearby coding gene CHD2 - early-onset syndrome of severe encephalopathy with cortical atrophy, cerebral hypomyelination, & facial dysmorphism Clinical manifestations: - one patient Emma - wheelchair-bound - non-verbal - uses a feeding tube - severe intellectual delays

General

neurologic disease chromosomal deletion

References

  1. George J First Cases of New Neurology Syndrome Identified Long non-coding RNA implicated in human disease MedPage Today October 23, 2024 https://www.medpagetoday.com/neurology/generalneurology/112548 - Ganesh VS, Riquin K, Chatron N et al Neurodevelopmental Disorder Caused by Deletion of CHASERR, a lncRNA Gene. N Engl J Med. 2024 Oct 24;391(16):1511-1518. PMID: 39442041