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Bardet-Biedl syndrome(BBS)

MedGen UID:
156019
Concept ID:
C0752166
Disease or Syndrome
Synonym: BBS
SNOMED CT: Bardet-Biedl syndrome (5619004); LMBB - Laurence-Moon-Bardet-Biedl syndrome (5619004); Biedl-Bardet syndrome (5619004)
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).
Oligogenic inheritance
MedGen UID:
253949
Concept ID:
C1136026
Genetic Function
Source: Orphanet
A type of multifactorial inheritance governed by the simultaneous action of a few gene loci. It is recommended this term be used for traits governed by three loci, although it is noted that usage of this term in the literature is not uniform.
 
Genes (locations): BBS1 (11q13.2); BBS4 (15q24.1); MKKS (20p12.2); SDCCAG8 (1q43-44)
Related genes: BBS12, BBS5, TTC8, TMEM67, ARL6, CEP290, BBS10, CCDC28B, BBS7, MKS1, WDPCP, BBS9, TRIM32, BBS2
 
Monarch Initiative: MONDO:0015229
OMIM® Phenotypic series: PS209900
Orphanet: ORPHA110

Definition

Bardet-Biedl syndrome is a disorder that affects many parts of the body. The signs and symptoms of this condition vary among affected individuals, even among members of the same family.

Vision loss is one of the major features of Bardet-Biedl syndrome. Loss of vision occurs as the light-sensing tissue at the back of the eye (the retina) gradually deteriorates. Problems with night vision become apparent by mid-childhood, followed by blind spots that develop in the side (peripheral) vision. Over time, these blind spots enlarge and merge to produce tunnel vision. Most people with Bardet-Biedl syndrome also develop blurred central vision (poor visual acuity) and become legally blind by adolescence or early adulthood.

Obesity is another characteristic feature of Bardet-Biedl syndrome. Abnormal weight gain typically begins in early childhood and continues to be an issue throughout life. Complications of obesity can include type 2 diabetes, high blood pressure (hypertension), and abnormally high cholesterol levels (hypercholesterolemia).

Other major signs and symptoms of Bardet-Biedl syndrome include the presence of extra fingers or toes (polydactyly), intellectual disability or learning problems, and abnormalities of the genitalia. Most affected males produce reduced amounts of sex hormones (hypogonadism), and they are usually unable to father biological children (infertile). Many people with Bardet-Biedl syndrome also have kidney abnormalities, which can be serious or life-threatening.

Additional features of Bardet-Biedl syndrome can include impaired speech, delayed development of motor skills such as standing and walking, behavioral problems such as emotional immaturity and inappropriate outbursts, and clumsiness or poor coordination. Distinctive facial features, dental abnormalities, unusually short or fused fingers or toes, and a partial or complete loss of the sense of smell (anosmia) have also been reported in some people with Bardet-Biedl syndrome. Additionally, this condition can affect the heart, liver, and digestive system. [from MedlinePlus Genetics]

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
Spahiu L, Behluli E, Grajçevci-Uka V, Liehr T, Temaj G
J Mother Child 2022 Mar 1;26(1):118-123. Epub 2023 Feb 22 doi: 10.34763/jmotherandchild.20222601.d-22-00034. PMID: 36803942Free 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

Curated

Slavotinek A, Beales P
Eur J Hum Genet 2011 Mar;19(3) Epub 2010 Dec 8 doi: 10.1038/ejhg.2010.199. PMID: 21150877Free PMC Article

Recent clinical studies

Etiology

De Groof J, Dachy A, Breysem L, Mekahli D
Arch Pediatr 2023 May;30(4):240-246. Epub 2023 Apr 14 doi: 10.1016/j.arcped.2023.02.005. PMID: 37062654
Travor MD
Methods Mol Biol 2023;2560:181-215. doi: 10.1007/978-1-0716-2651-1_19. PMID: 36481897
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
Littleton SH, Berkowitz RI, Grant SFA
Mol Diagn Ther 2020 Dec;24(6):653-663. Epub 2020 Oct 1 doi: 10.1007/s40291-020-00496-1. PMID: 33006084Free 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

Diagnosis

De Groof J, Dachy A, Breysem L, Mekahli D
Arch Pediatr 2023 May;30(4):240-246. Epub 2023 Apr 14 doi: 10.1016/j.arcped.2023.02.005. PMID: 37062654
Spahiu L, Behluli E, Grajçevci-Uka V, Liehr T, Temaj G
J Mother Child 2022 Mar 1;26(1):118-123. Epub 2023 Feb 22 doi: 10.34763/jmotherandchild.20222601.d-22-00034. PMID: 36803942Free PMC Article
Ni X, Shi T
Sci China Life Sci 2017 Jul;60(7):681-685. Epub 2017 Jun 14 doi: 10.1007/s11427-017-9100-1. PMID: 28623543
Antosik K, Borowiec M
Arch Immunol Ther Exp (Warsz) 2016 Dec;64(Suppl 1):157-160. Epub 2017 Jan 12 doi: 10.1007/s00005-016-0432-8. PMID: 28083605
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

Haqq AM, Chung WK, Dollfus H, Haws RM, Martos-Moreno GÁ, Poitou C, Yanovski JA, Mittleman RS, Yuan G, Forsythe E, Clément K, Argente J
Lancet Diabetes Endocrinol 2022 Dec;10(12):859-868. Epub 2022 Nov 7 doi: 10.1016/S2213-8587(22)00277-7. PMID: 36356613Free PMC Article
Kousi M, Söylemez O, Ozanturk A, Mourtzi N, Akle S, Jungreis I, Muller J, Cassa CA, Brand H, Mokry JA, Wolf MY, Sadeghpour A, McFadden K, Lewis RA, Talkowski ME, Dollfus H, Kellis M, Davis EE, Sunyaev SR, Katsanis N
Nat Genet 2020 Nov;52(11):1145-1150. Epub 2020 Oct 12 doi: 10.1038/s41588-020-0707-1. PMID: 33046855Free PMC Article
Chan S, Freund PR, MacDonald I
Curr Opin Pediatr 2013 Dec;25(6):645-52. doi: 10.1097/MOP.0000000000000026. PMID: 24126856
Zlotogora J
Hum Genet 2010 Nov;128(5):473-9. Epub 2010 Sep 18 doi: 10.1007/s00439-010-0890-8. PMID: 20852892
Farooqi IS
Best Pract Res Clin Endocrinol Metab 2005 Sep;19(3):359-74. doi: 10.1016/j.beem.2005.04.004. PMID: 16150380

Prognosis

De Groof J, Dachy A, Breysem L, Mekahli D
Arch Pediatr 2023 May;30(4):240-246. Epub 2023 Apr 14 doi: 10.1016/j.arcped.2023.02.005. PMID: 37062654
Best S, Yu J, Lord J, Roche M, Watson CM, Bevers RPJ, Stuckey A, Madhusudhan S, Jewell R, Sisodiya SM, Lin S, Turner S, Robinson H, Leslie JS, Baple E; Genomics England Research Consortium, Toomes C, Inglehearn C, Wheway G, Johnson CA
J Med Genet 2022 Dec;59(12):1151-1164. Epub 2022 Jun 28 doi: 10.1136/jmedgenet-2022-108476. PMID: 35764379Free PMC Article
Coppieters F, Lefever S, Leroy BP, De Baere E
Hum Mutat 2010 Oct;31(10):1097-108. doi: 10.1002/humu.21337. PMID: 20690115
Baker K, Beales PL
Am J Med Genet C Semin Med Genet 2009 Nov 15;151C(4):281-95. doi: 10.1002/ajmg.c.30231. PMID: 19876933
Walley AJ, Blakemore AI, Froguel P
Hum Mol Genet 2006 Oct 15;15 Spec No 2:R124-30. doi: 10.1093/hmg/ddl215. PMID: 16987875

Clinical prediction guides

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
Zlotogora J
Hum Genet 2010 Nov;128(5):473-9. Epub 2010 Sep 18 doi: 10.1007/s00439-010-0890-8. PMID: 20852892
Coppieters F, Lefever S, Leroy BP, De Baere E
Hum Mutat 2010 Oct;31(10):1097-108. doi: 10.1002/humu.21337. PMID: 20690115
Baker K, Beales PL
Am J Med Genet C Semin Med Genet 2009 Nov 15;151C(4):281-95. doi: 10.1002/ajmg.c.30231. PMID: 19876933
Pazour GJ
Curr Biol 2004 Jul 27;14(14):R575-7. doi: 10.1016/j.cub.2004.07.017. PMID: 15268880

Recent systematic reviews

Sonner S, Reilly K, Woolf AS, Chandler N, Kilby MD, Maher ER, Flanagan C, McKnight AJ, Mone F
Prenat Diagn 2024 Feb;44(2):187-195. Epub 2023 Dec 6 doi: 10.1002/pd.6479. PMID: 38056891
Deng L, Liu Y, Yuan M, Meng M, Yang Y, Sun L
Clin Chim Acta 2022 Mar 1;528:16-28. Epub 2022 Jan 20 doi: 10.1016/j.cca.2022.01.012. PMID: 35065907

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