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Ciliopathy

MedGen UID:
908923
Concept ID:
C4277690
Congenital Abnormality
Synonym: Ciliopathies
 
Monarch Initiative: MONDO:0005308
Orphanet: ORPHA363250

Definition

A genetic disorder of the cellular cilia or the cilia anchoring structures, the basal bodies, or of ciliary function. [from MONDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVCiliopathy

Professional guidelines

PubMed

Han JC, Rasmussen MC, Forte AR, Schrage SB, Zafar SK, Haqq AM
Gastroenterol Clin North Am 2023 Dec;52(4):733-750. Epub 2023 Sep 27 doi: 10.1016/j.gtc.2023.08.005. PMID: 37919024
Gana S, Serpieri V, Valente EM
Am J Med Genet C Semin Med Genet 2022 Mar;190(1):72-88. Epub 2022 Mar 3 doi: 10.1002/ajmg.c.31963. PMID: 35238134Free PMC Article
Raina R, Chakraborty R, Sethi SK, Kumar D, Gibson K, Bergmann C
Am J Kidney Dis 2021 Jul;78(1):125-141. Epub 2021 Jan 6 doi: 10.1053/j.ajkd.2020.10.021. PMID: 33418012

Recent clinical studies

Etiology

Raidt J, Loges NT, Olbrich H, Wallmeier J, Pennekamp P, Omran H
Presse Med 2023 Sep;52(3):104171. Epub 2023 Jul 27 doi: 10.1016/j.lpm.2023.104171. PMID: 37516247
Gana S, Serpieri V, Valente EM
Am J Med Genet C Semin Med Genet 2022 Mar;190(1):72-88. Epub 2022 Mar 3 doi: 10.1002/ajmg.c.31963. PMID: 35238134Free PMC Article
Senum SR, Li YSM, Benson KA, Joli G, Olinger E, Lavu S, Madsen CD, Gregory AV, Neatu R, Kline TL, Audrézet MP, Outeda P, Nau CB, Meijer E, Ali H, Steinman TI, Mrug M, Phelan PJ, Watnick TJ, Peters DJM, Ong ACM, Conlon PJ, Perrone RD, Cornec-Le Gall E, Hogan MC, Torres VE, Sayer JA; Genomics England Research Consortium, the HALT PKD, CRISP, DIPAK, ADPKD Modifier, and TAME PKD studies, Harris PC
Am J Hum Genet 2022 Jan 6;109(1):136-156. Epub 2021 Dec 9 doi: 10.1016/j.ajhg.2021.11.016. PMID: 34890546Free PMC Article
McConnachie DJ, Stow JL, Mallett AJ
Am J Kidney Dis 2021 Mar;77(3):410-419. Epub 2020 Oct 9 doi: 10.1053/j.ajkd.2020.08.012. PMID: 33039432
Wallmeier J, Nielsen KG, Kuehni CE, Lucas JS, Leigh MW, Zariwala MA, Omran H
Nat Rev Dis Primers 2020 Sep 17;6(1):77. doi: 10.1038/s41572-020-0209-6. PMID: 32943623

Diagnosis

Raidt J, Loges NT, Olbrich H, Wallmeier J, Pennekamp P, Omran H
Presse Med 2023 Sep;52(3):104171. Epub 2023 Jul 27 doi: 10.1016/j.lpm.2023.104171. PMID: 37516247
Tian X, Zhao H, Zhou J
Elife 2023 Jul 19;12 doi: 10.7554/eLife.87623. PMID: 37466224Free PMC Article
Gana S, Serpieri V, Valente EM
Am J Med Genet C Semin Med Genet 2022 Mar;190(1):72-88. Epub 2022 Mar 3 doi: 10.1002/ajmg.c.31963. PMID: 35238134Free PMC Article
Wallmeier J, Nielsen KG, Kuehni CE, Lucas JS, Leigh MW, Zariwala MA, Omran H
Nat Rev Dis Primers 2020 Sep 17;6(1):77. doi: 10.1038/s41572-020-0209-6. PMID: 32943623
Forsythe E, Beales PL
Eur J Hum Genet 2013 Jan;21(1):8-13. Epub 2012 Jun 20 doi: 10.1038/ejhg.2012.115. PMID: 22713813Free PMC Article

Therapy

Paff T, Omran H, Nielsen KG, Haarman EG
Int J Mol Sci 2021 Sep 11;22(18) doi: 10.3390/ijms22189834. PMID: 34575997Free PMC Article
Kaur P, Chaudhry C, Neelam H, Panigrahi I
BMJ Case Rep 2021 Jan 28;14(1) doi: 10.1136/bcr-2020-236325. PMID: 33509858Free PMC Article
Yeung JC, Katwa UA, Lee GS
Int J Pediatr Otorhinolaryngol 2017 Nov;102:127-132. Epub 2017 Sep 12 doi: 10.1016/j.ijporl.2017.09.004. PMID: 29106859
Haws RM, Joshi A, Shah SA, Alkandari O, Turman MA
Pediatr Nephrol 2016 Nov;31(11):2153-61. Epub 2016 Jun 1 doi: 10.1007/s00467-016-3415-4. PMID: 27245600
Deschler EK, Sun JK, Silva PS
Semin Ophthalmol 2014 Sep-Nov;29(5-6):290-300. doi: 10.3109/08820538.2014.959198. PMID: 25325854

Prognosis

Bannell TAK, Cockburn JJB
Ann Hum Genet 2024 Jan;88(1):58-75. Epub 2023 Oct 31 doi: 10.1111/ahg.12535. PMID: 37905714
Gupta N, D'Acierno M, Zona E, Capasso G, Zacchia M
Am J Med Genet C Semin Med Genet 2022 Mar;190(1):9-19. Epub 2022 Apr 4 doi: 10.1002/ajmg.c.31970. PMID: 35373910Free PMC Article
Gana S, Serpieri V, Valente EM
Am J Med Genet C Semin Med Genet 2022 Mar;190(1):72-88. Epub 2022 Mar 3 doi: 10.1002/ajmg.c.31963. PMID: 35238134Free PMC Article
Luo F, Tao YH
Nephrology (Carlton) 2018 Oct;23(10):904-911. Epub 2018 Jun 21 doi: 10.1111/nep.13393. PMID: 29717526Free PMC Article
Yeung JC, Katwa UA, Lee GS
Int J Pediatr Otorhinolaryngol 2017 Nov;102:127-132. Epub 2017 Sep 12 doi: 10.1016/j.ijporl.2017.09.004. PMID: 29106859

Clinical prediction guides

Kim AH, Sakin I, Viviano S, Tuncel G, Aguilera SM, Goles G, Jeffries L, Ji W, Lakhani SA, Kose CC, Silan F, Oner SS, Kaplan OI; MarmaRare Group, Ergoren MC, Mishra-Gorur K, Gunel M, Sag SO, Temel SG, Deniz E
Life Sci Alliance 2024 Oct;7(10) Epub 2024 Aug 21 doi: 10.26508/lsa.202402708. PMID: 39168639Free PMC Article
McDonald A, Wijnholds J
Int J Mol Sci 2024 Mar 1;25(5) doi: 10.3390/ijms25052887. PMID: 38474133Free PMC Article
Bannell TAK, Cockburn JJB
Ann Hum Genet 2024 Jan;88(1):58-75. Epub 2023 Oct 31 doi: 10.1111/ahg.12535. PMID: 37905714
Bachmann-Gagescu R, Dempsey JC, Phelps IG, O'Roak BJ, Knutzen DM, Rue TC, Ishak GE, Isabella CR, Gorden N, Adkins J, Boyle EA, de Lacy N, O'Day D, Alswaid A, Ramadevi A R, Lingappa L, Lourenço C, Martorell L, Garcia-Cazorla À, Ozyürek H, Haliloğlu G, Tuysuz B, Topçu M; University of Washington Center for Mendelian Genomics, Chance P, Parisi MA, Glass IA, Shendure J, Doherty D
J Med Genet 2015 Aug;52(8):514-22. Epub 2015 Jun 19 doi: 10.1136/jmedgenet-2015-103087. PMID: 26092869Free PMC Article
Forsythe E, Beales PL
Eur J Hum Genet 2013 Jan;21(1):8-13. Epub 2012 Jun 20 doi: 10.1038/ejhg.2012.115. PMID: 22713813Free PMC Article

Recent systematic reviews

Vrabič N, Fakin A, Tekavčič Pompe M
J Fr Ophtalmol 2024 Oct;47(8):104232. Epub 2024 Aug 29 doi: 10.1016/j.jfo.2024.104232. PMID: 39213781
Junior JHMF, Junior SP, Pustilnik HN, de Almeida Leão L, da Paz MGDS, Araujo TB, de Deus FOG, Alcântara T, Dourado JC, de Avellar LM
Childs Nerv Syst 2024 Jul;40(7):2161-2168. Epub 2024 Mar 9 doi: 10.1007/s00381-024-06346-3. PMID: 38459147
Bea-Mascato B, Valverde D
J Med Genet 2023 Dec 21;61(1):18-26. doi: 10.1136/jmg-2023-109175. PMID: 37321834Free PMC Article
Hasanain AA, Soliman MAR, Elwy R, Ezzat AAM, Abdel-Bari SH, Marx S, Jenkins A, El Refaee E, Zohdi A
Br J Neurosurg 2022 Jun;36(3):329-339. Epub 2022 May 17 doi: 10.1080/02688697.2022.2074373. PMID: 35579079
Rim JH, Choi YJ, Gee HY
Biomolecules 2020 Mar 13;10(3) doi: 10.3390/biom10030449. PMID: 32183147Free PMC Article

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