Login / Signup

Uniparental Disomy of Chromosome 4: A Case of Whole Chromosome UPD Presenting with LRBA Deficiency.

Bilgesu AkErhan ParıltayReyhan GümüşburunCeyda Tunakan DalgıçAyça AykutAsude DurmazHaluk AkınÖmür ArdenizBernice Lo
Published in: Journal of clinical immunology (2024)
In conclusion, this case study underscores the critical role of LRBA in immune regulation and highlights the clinical heterogeneity associated with LRBA deficiency. The patient's presentation with severe immune dysregulation, including massive splenomegaly, portal hypertension, and the novel finding of mast cell-derived interstitial cystitis, expands the clinical spectrum of LRBA mutations. The identification of an apparently homozygous LRBA mutation via next-generation sequencing further emphasizes the importance of genetic analysis in diagnosing monogenic defects manifested as CVID-like phenotype. This is the first reported case of LRBA deficiency due to whole chromosome UPD to our knowledge. Future research should focus on elucidating the full range of clinical manifestations and developing targeted therapies for patients with LRBA deficiency.
Keyphrases
  • copy number
  • case report
  • blood pressure
  • healthcare
  • early onset
  • gene expression
  • dna methylation
  • current status
  • cell free