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Renal dysplasia and retinal aplasia(SLSN)

MedGen UID:
96045
Concept ID:
C0403553
Disease or Syndrome
Synonyms: Renal dysplasia retinal aplasia; Senior-Loken syndrome; SLSN
SNOMED CT: Renal dysplasia and retinal aplasia (236531005); Loken Senior syndrome (236531005)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Related genes: NPHP4, CEP290, WDR19, TRAF3IP1, SDCCAG8, IQCB1, NPHP1
 
Monarch Initiative: MONDO:0017842
OMIM®: 266900
OMIM® Phenotypic series: PS266900
Orphanet: ORPHA3156

Definition

Senior-Loken syndrome is an autosomal recessive disease with the main features of nephronophthisis (NPHP; see 256100) and Leber congenital amaurosis (see 204000). Mutations in some of the same genes that cause nephronophthisis (see 256100) cause Senior-Loken syndrome. Genetic Heterogeneity of Senior-Loken Syndrome Other forms of SLSN include SLSN4 (606996), caused by mutation in the NPHP4 gene (607215) on chromosome 1p36; SLSN5 (609254), caused by mutation in the NPHP5 gene (IQCB1; 609237) on chromosome 3q13; SLSN6 (610189), caused by mutation in the NPHP6 gene (CEP290; 610142) on chromosome 12q21; SLSN7 (613615), caused by mutation in the SDCCAG8 gene (613524) on chromosome 1q43; SLSN8 (616307), caused by mutation in the WDR19 gene (608151) on chromosome 4p14; and SLSN9 (616629), caused by mutation in the TRAF3IP1 gene (607380) on chromosome 2q37. Another form of SLSN, SLSN3 (606995), has been mapped to a locus on chromosome 3q22, overlapping the NPHP3 locus (604387). [from OMIM]

Professional guidelines

PubMed

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Recent clinical studies

Etiology

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
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Diagnosis

Tay SA, Vincent AL
Ophthalmic Genet 2020 Aug;41(4):354-357. Epub 2020 May 20 doi: 10.1080/13816810.2020.1766086. PMID: 32432520
Kang HG, Lee HK, Ahn YH, Joung JG, Nam J, Kim NK, Ko JM, Cho MH, Shin JI, Kim J, Park HW, Park YS, Ha IS, Chung WY, Lee DY, Kim SY, Park WY, Cheong HI
Exp Mol Med 2016 Aug 5;48(8):e251. doi: 10.1038/emm.2016.63. PMID: 27491411Free PMC Article
Valente EM, Dallapiccola B, Bertini E
Handb Clin Neurol 2013;113:1879-88. doi: 10.1016/B978-0-444-59565-2.00058-7. PMID: 23622411
Simms RJ, Eley L, Sayer JA
Eur J Hum Genet 2009 Apr;17(4):406-16. Epub 2008 Dec 10 doi: 10.1038/ejhg.2008.238. PMID: 19066617Free PMC Article
Konrad M, Saunier S, Calado J, Gubler MC, Broyer M, Antignac C
J Mol Med (Berl) 1998 Apr;76(5):310-6. doi: 10.1007/s001090050222. PMID: 9587065

Therapy

Bartiromo M, Borchi B, Botta A, Bagalà A, Lugli G, Tilli M, Cavallo A, Xhaferi B, Cutruzzulà R, Vaglio A, Bresci S, Larti A, Bartoloni A, Cirami C
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Al Zahrani Y, Peck D
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Prognosis

Tay SA, Vincent AL
Ophthalmic Genet 2020 Aug;41(4):354-357. Epub 2020 May 20 doi: 10.1080/13816810.2020.1766086. PMID: 32432520
König J, Kranz B, König S, Schlingmann KP, Titieni A, Tönshoff B, Habbig S, Pape L, Häffner K, Hansen M, Büscher A, Bald M, Billing H, Schild R, Walden U, Hampel T, Staude H, Riedl M, Gretz N, Lablans M, Bergmann C, Hildebrandt F, Omran H, Konrad M; Gesellschaft für Pädiatrische Nephrologie (GPN)
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Clinical prediction guides

Leggatt G, Cheng G, Narain S, Briseño-Roa L, Annereau JP; Genomics England Research Consortium, Gast C, Gilbert RD, Ennis S
Sci Rep 2023 Jun 9;13(1):9369. doi: 10.1038/s41598-023-32169-4. PMID: 37296294Free PMC Article
Wang J, Li S, Jiang Y, Wang Y, Ouyang J, Yi Z, Sun W, Jia X, Xiao X, Wang P, Zhang Q
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König J, Kranz B, König S, Schlingmann KP, Titieni A, Tönshoff B, Habbig S, Pape L, Häffner K, Hansen M, Büscher A, Bald M, Billing H, Schild R, Walden U, Hampel T, Staude H, Riedl M, Gretz N, Lablans M, Bergmann C, Hildebrandt F, Omran H, Konrad M; Gesellschaft für Pädiatrische Nephrologie (GPN)
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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

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