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Abnormal protein N-linked glycosylation

MedGen UID:
868545
Concept ID:
C4022944
Finding
HPO: HP:0012347

Definition

An anomaly of protein N-linked glycosylation, i.e., an abnormality of the protein glycosylation process in which a carbohydrate or carbohydrate derivative unit is added to a protein via a nitrogen atom in an amino acid residue in a protein. [from HPO]

Conditions with this feature

TMEM199-CDG
MedGen UID:
895025
Concept ID:
C4225190
Disease or Syndrome
Congenital disorder of glycosylation type IIp (CDG2P) is an autosomal recessive metabolic disorder characterized by mild liver dysfunction, which may be found incidentally during adolescence. Laboratory abnormalities include elevated liver enzymes and alkaline phosphatase, coagulation factor deficiencies, hypercholesterolemia, and low ceruloplasmin. Serum isoelectric focusing of proteins shows a combined defect of N- and O-glycosylation, suggestive of a Golgi defect (summary by Jansen et al., 2016). For an overview of congenital disorders of glycosylation, see CDG1A (212065) and CDG2A (212066).
Immunodeficiency 47
MedGen UID:
934786
Concept ID:
C4310819
Disease or Syndrome
Immunodeficiency-47 (IMD47) is an X-linked recessive complex syndrome characterized by liver dysfunction, recurrent bacterial infections, hypogammaglobulinemia, and defective glycosylation of serum proteins. Some patients also have neurologic abnormalities (summary by Jansen et al., 2016).
Congenital disorder of glycosylation, type 2v
MedGen UID:
1794181
Concept ID:
C5561971
Disease or Syndrome
Congenital disorder of glycosylation type 2v (CDG2V) is an autosomal recessive disorder characterized by neurodevelopmental delay and variable facial dysmorphisms (Polla et al., 2021).
Congenital disorder of glycosylation, type IIw
MedGen UID:
1794196
Concept ID:
C5561986
Disease or Syndrome
Congenital disorder of glycosylation type IIw (CDG2W) is an autosomal dominant metabolic disorder characterized by liver dysfunction, coagulation deficiencies, and profound abnormalities in N-glycosylation of serum specific proteins. All reported patients carry the same mutation (602671.0017) (summary by Ng et al., 2021). For an overview of congenital disorders of glycosylation, see CDG1A (212065) and CDG2A (212066).

Recent clinical studies

Etiology

Wilson MP, Durin Z, Unal Ö, Ng BG, Marrecau T, Keldermans L, Souche E, Rymen D, Gündüz M, Köse G, Sturiale L, Garozzo D, Freeze HH, Jaeken J, Foulquier F, Matthijs G
Hum Mol Genet 2022 Aug 17;31(15):2571-2581. doi: 10.1093/hmg/ddac055. PMID: 35262690Free PMC Article
Davids M, Kane MS, He M, Wolfe LA, Li X, Raihan MA, Chao KR, Bone WP, Boerkoel CF, Gahl WA, Toro C
J Med Genet 2016 Mar;53(3):180-9. Epub 2015 Dec 14 doi: 10.1136/jmedgenet-2015-103338. PMID: 26668131Free PMC Article
Li X, Raihan MA, Reynoso FJ, He M
Curr Protoc Hum Genet 2015 Jul 1;86:17.18.1-17.18.22. doi: 10.1002/0471142905.hg1718s86. PMID: 26132001
Tucholski J, Simmons MS, Pinner AL, McMillan LD, Haroutunian V, Meador-Woodruff JH
Neuroreport 2013 Aug 21;24(12):688-91. doi: 10.1097/WNR.0b013e328363bd8a. PMID: 23820740Free PMC Article
Tucholski J, Simmons MS, Pinner AL, Haroutunian V, McCullumsmith RE, Meador-Woodruff JH
Schizophr Res 2013 May;146(1-3):177-83. Epub 2013 Feb 23 doi: 10.1016/j.schres.2013.01.031. PMID: 23462048Free PMC Article

Diagnosis

Ng BG, Freeze HH, Himmelreich N, Blau N, Ferreira CR
Mol Genet Metab 2024 May;142(1):108476. Epub 2024 Apr 10 doi: 10.1016/j.ymgme.2024.108476. PMID: 38653092Free PMC Article
Alsharhan H, He M, Edmondson AC, Daniel EJP, Chen J, Donald T, Bakhtiari S, Amor DJ, Jones EA, Vassallo G, Vincent M, Cogné B, Deb W, Werners AH, Jin SC, Bilguvar K, Christodoulou J, Webster RI, Yearwood KR, Ng BG, Freeze HH, Kruer MC, Li D, Raymond KM, Bhoj EJ, Sobering AK
J Inherit Metab Dis 2021 Jul;44(4):1001-1012. Epub 2021 Mar 26 doi: 10.1002/jimd.12378. PMID: 33734437Free PMC Article
Scott K, Gadomski T, Kozicz T, Morava E
J Inherit Metab Dis 2014 Jul;37(4):609-17. Epub 2014 May 15 doi: 10.1007/s10545-014-9720-9. PMID: 24831587Free PMC Article
Tucholski J, Simmons MS, Pinner AL, Haroutunian V, McCullumsmith RE, Meador-Woodruff JH
Schizophr Res 2013 May;146(1-3):177-83. Epub 2013 Feb 23 doi: 10.1016/j.schres.2013.01.031. PMID: 23462048Free PMC Article
Kościelak J
Acta Biochim Pol 1999;46(3):727-38. PMID: 10698281

Therapy

Verdura S, Cuyàs E, Cortada E, Brunet J, Lopez-Bonet E, Martin-Castillo B, Bosch-Barrera J, Encinar JA, Menendez JA
Aging (Albany NY) 2020 Jan 4;12(1):8-34. doi: 10.18632/aging.102646. PMID: 31901900Free PMC Article
Höti N, Yang S, Hu Y, Shah P, Haffner MC, Zhang H
Prostate Cancer Prostatic Dis 2018 Apr;21(1):137-146. Epub 2018 Jan 16 doi: 10.1038/s41391-017-0016-7. PMID: 29339807Free PMC Article
Morelle W, Potelle S, Witters P, Wong S, Climer L, Lupashin V, Matthijs G, Gadomski T, Jaeken J, Cassiman D, Morava E, Foulquier F
J Clin Endocrinol Metab 2017 Apr 1;102(4):1375-1386. doi: 10.1210/jc.2016-3443. PMID: 28323990Free PMC Article
Kippe JM, Mueller TM, Haroutunian V, Meador-Woodruff JH
Schizophr Res 2015 Aug;166(1-3):219-24. Epub 2015 Jun 20 doi: 10.1016/j.schres.2015.06.002. PMID: 26104473Free PMC Article
Kościelak J
Acta Biochim Pol 1999;46(3):727-38. PMID: 10698281

Prognosis

Alharbi H, Daniel EJP, Thies J, Chang I, Goldner DL, Ng BG, Witters P, Aqul A, Velez-Bartolomei F, Enns GM, Hsu E, Kichula E, Lee E, Lourenco C, Poskanzer SA, Rasmussen S, Saarela K, Wang YM, Raymond KM, Schultz MJ, Freeze HH, Lam C, Edmondson AC, He M
J Inherit Metab Dis 2023 Mar;46(2):300-312. Epub 2023 Jan 29 doi: 10.1002/jimd.12589. PMID: 36651831Free PMC Article
Alsharhan H, He M, Edmondson AC, Daniel EJP, Chen J, Donald T, Bakhtiari S, Amor DJ, Jones EA, Vassallo G, Vincent M, Cogné B, Deb W, Werners AH, Jin SC, Bilguvar K, Christodoulou J, Webster RI, Yearwood KR, Ng BG, Freeze HH, Kruer MC, Li D, Raymond KM, Bhoj EJ, Sobering AK
J Inherit Metab Dis 2021 Jul;44(4):1001-1012. Epub 2021 Mar 26 doi: 10.1002/jimd.12378. PMID: 33734437Free PMC Article
Amor DJ, Stephenson SEM, Mustapha M, Mensah MA, Ockeloen CW, Lee WS, Tankard RM, Phelan DG, Shinawi M, de Brouwer APM, Pfundt R, Dowling C, Toler TL, Sutton VR, Agolini E, Rinelli M, Capolino R, Martinelli D, Zampino G, Dumić M, Reardon W, Shaw-Smith C, Leventer RJ, Delatycki MB, Kleefstra T, Mundlos S, Mortier G, Bahlo M, Allen NJ, Lockhart PJ
Am J Hum Genet 2019 May 2;104(5):914-924. Epub 2019 Apr 11 doi: 10.1016/j.ajhg.2019.02.026. PMID: 30982611Free PMC Article
Maratha A, Stockmann H, Coss KP, Estela Rubio-Gozalbo M, Knerr I, Fitzgibbon M, McVeigh TP, Foley P, Moss C, Colhoun HO, van Erven B, Stephens K, Doran P, Rudd P, Treacy E
Eur J Hum Genet 2016 Jul;24(7):976-84. Epub 2016 Jan 6 doi: 10.1038/ejhg.2015.254. PMID: 26733289Free PMC Article
Liu Y, Xia B, Gleason TJ, Castañeda U, He M, Berry GT, Fridovich-Keil JL
Mol Genet Metab 2012 Aug;106(4):442-54. Epub 2012 Jun 12 doi: 10.1016/j.ymgme.2012.05.025. PMID: 22743281Free PMC Article

Clinical prediction guides

Ligezka AN, Budhraja R, Nishiyama Y, Fiesel FC, Preston G, Edmondson A, Ranatunga W, Van Hove JLK, Watzlawik JO, Springer W, Pandey A, Morava E, Kozicz T
Genes (Basel) 2023 Aug 4;14(8) doi: 10.3390/genes14081585. PMID: 37628636Free PMC Article
Balakrishnan B, Altassan R, Budhraja R, Liou W, Lupo A, Bryant S, Mankouski A, Radenkovic S, Preston GJ, Pandey A, Boudina S, Kozicz T, Morava E, Lai K
Transl Res 2023 Jul;257:1-14. Epub 2023 Jan 26 doi: 10.1016/j.trsl.2023.01.004. PMID: 36709920Free PMC Article
Amor DJ, Stephenson SEM, Mustapha M, Mensah MA, Ockeloen CW, Lee WS, Tankard RM, Phelan DG, Shinawi M, de Brouwer APM, Pfundt R, Dowling C, Toler TL, Sutton VR, Agolini E, Rinelli M, Capolino R, Martinelli D, Zampino G, Dumić M, Reardon W, Shaw-Smith C, Leventer RJ, Delatycki MB, Kleefstra T, Mundlos S, Mortier G, Bahlo M, Allen NJ, Lockhart PJ
Am J Hum Genet 2019 May 2;104(5):914-924. Epub 2019 Apr 11 doi: 10.1016/j.ajhg.2019.02.026. PMID: 30982611Free PMC Article
Maratha A, Stockmann H, Coss KP, Estela Rubio-Gozalbo M, Knerr I, Fitzgibbon M, McVeigh TP, Foley P, Moss C, Colhoun HO, van Erven B, Stephens K, Doran P, Rudd P, Treacy E
Eur J Hum Genet 2016 Jul;24(7):976-84. Epub 2016 Jan 6 doi: 10.1038/ejhg.2015.254. PMID: 26733289Free PMC Article
Liu Y, Xia B, Gleason TJ, Castañeda U, He M, Berry GT, Fridovich-Keil JL
Mol Genet Metab 2012 Aug;106(4):442-54. Epub 2012 Jun 12 doi: 10.1016/j.ymgme.2012.05.025. PMID: 22743281Free PMC Article

Recent systematic reviews

Li Y, Ding B, Wang X, Ding Q
Thromb Res 2022 Sep;217:36-47. Epub 2022 Jul 14 doi: 10.1016/j.thromres.2022.07.005. PMID: 35853369

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