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Rare heterozygous loss-of-function events in NRXN3 have been implicated in autism spectrum disorder through limited family and sporadic case studies. In a three-generation Chinese pedigree, a rare exonic deletion in the NRXN3 alpha isoform co-segregated with autism, moderate intellectual disability (HP:0002342), attention-deficit hyperactivity disorder (HP:0007018), and variable neuropsychiatric features across three affected carriers ([PMID:30076746]). A literature compilation of 23 sporadic deletion cases revealed autism features in 9 individuals (39%) ([PMID:30076746]).
Further support comes from a heterozygous frameshift variant c.159_160del (p.Phe53LeufsTer16) in the NRXN3 beta isoform identified in a 5-year-old girl with autism spectrum disorder and developmental delay; this variant was inherited from an unaffected mother, consistent with incomplete penetrance of NRXN3 haploinsufficiency ([PMID:37372397]). Functional assays to date demonstrate complex splicing and synaptic localization of neurexin-3 but lack autism-specific mechanistic studies.
These data meet a Limited level of clinical validity for NRXN3 in autism due to small cohort sizes, incomplete penetrance, and absence of replication in large independent studies. Further large-scale genetic screening and targeted functional assays are needed to establish NRXN3 as a diagnostic marker for ASD.
Key Take-home: NRXN3 haploinsufficiency shows preliminary evidence for an autosomal dominant contribution to autism with incomplete penetrance, but current data remain limited for routine clinical application.
Gene–Disease AssociationLimitedSingle multiplex pedigree with three carriers co-segregating an NRXN3 exonic deletion; 9/23 sporadic deletion cases with autism features; limited cohort size and replication Genetic EvidenceLimitedOne family with segregation in 3 individuals; additional sporadic deletion cases (9/23) with autism features; one inherited frameshift variant described Functional EvidenceLimitedExpression and splicing studies demonstrate synaptic role of NRXN3, but no direct autism-specific functional assays |