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Mendelian Disorders in an Interstitial Cystitis/Bladder Pain Syndrome Cohort.
Estrella, Elicia; Rockowitz, Shira; Thorne, Marielle; Smith, Pressley; Petit, Jeanette; Zehnder, Veronica; Yu, Richard N; Bauer, Stuart; Berde, Charles; Agrawal, Pankaj B; Beggs, Alan H; Gharavi, Ali G; Kunkel, Louis; Brownstein, Catherine A.
Afiliación
  • Estrella E; Department of Neurology Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Rockowitz S; Division of Genetics and Genomics Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Thorne M; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Smith P; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Petit J; Research Computing Information Technology Boston Children's Hospital Boston MA 02115 USA.
  • Zehnder V; Division of Genetics and Genomics Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Yu RN; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Bauer S; Division of Genetics and Genomics Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Berde C; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Agrawal PB; Division of Genetics and Genomics Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Beggs AH; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Gharavi AG; Division of Genetics and Genomics Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Kunkel L; The Manton Center for Orphan disease Research Boston Children's Hospital Harvard Medical School Boston MA 02115 USA.
  • Brownstein CA; Department of Urology Boston Children's Hospital Boston MA 02115 USA.
Adv Genet (Hoboken) ; 4(1): 2200013, 2023 Mar.
Article en En | MEDLINE | ID: mdl-36910591
Interstitial cystitis/bladder pain syndrome (IC/BPS) is a chronic pain disorder causing symptoms of urinary frequency, urgency, and bladder discomfort or pain. Although this condition affects a large population, little is known about its etiology. Genetic analyses of whole exome sequencing are performed on 109 individuals with IC/BPS. One family has a previously reported SIX5 variant (ENST00000317578.6:c.472G>A, p.Ala158Thr), consistent with Branchiootorenal syndrome 2 (BOR2). A likely pathogenic heterozygous variant in ATP2A2 (ENST00000539276.2:c.235G>A, p.Glu79Lys) is identified in two unrelated probands, indicating possible Darier-White disease. Two private heterozygous variants are identified in ATP2C1 (ENST00000393221.4:c.2358A>T, p.Glu786Asp (VUS/Likely Pathogenic) and ENST00000393221.4:c.989C>G, p.Thr330Ser (likely pathogenic)), indicative of Hailey-Hailey Disease. Sequence kernel association test analysis finds an increased burden of rare ATP2C1 variants in the IC/BPS cases versus a control cohort (p = 0.03, OR = 6.76), though does not survive Bonferroni correction. The data suggest that some individuals with IC/BPS may have unrecognized Mendelian syndromes. Comprehensive phenotyping and genotyping aid in understanding the range of diagnoses in the population-based IC/BPS cohort. Conversely, ATP2C1, ATP2A2, and SIX5 may be candidate genes for IC/BPS. Further evaluation with larger numbers is needed. Genetically screening individuals with IC/BPS may help diagnose and treat this painful disorder due to its heterogeneous nature.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Risk_factors_studies Idioma: En Revista: Adv Genet (Hoboken) Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Tipo de estudio: Risk_factors_studies Idioma: En Revista: Adv Genet (Hoboken) Año: 2023 Tipo del documento: Article Pais de publicación: Estados Unidos