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Ataxia-telangiectasia-like disorder(ATLD)

MedGen UID:
348929
Concept ID:
C1858391
Disease or Syndrome
Synonym: ATLD
SNOMED CT: Ataxia-telangiectasia-like disorder (700058006); ATLD - ataxia telangiectasia-like disorder (700058006)
 
Monarch Initiative: MONDO:0011457
OMIM®: 604391
OMIM® Phenotypic series: PS604391

Definition

An autosomal recessive condition caused by mutation(s) in the MRE11A gene, encoding double-strand break repair protein MRE11. It is characterized by progressive cerebellar degeneration resulting in ataxia and oculomotor apraxia. [from MONDO]

Recent clinical studies

Etiology

Worm C, Schambye MER, Mkrtchyan GV, Veviorskiy A, Shneyderman A, Ozerov IV, Zhavoronkov A, Bakula D, Scheibye-Knudsen M
Aging (Albany NY) 2024 Feb 9;16(3):2026-2046. doi: 10.18632/aging.205537. PMID: 38345566Free PMC Article
Unterholzner L
Immunobiology 2013 Nov;218(11):1312-21. Epub 2013 Jul 29 doi: 10.1016/j.imbio.2013.07.007. PMID: 23962476
Liu W, Narayanan V
Semin Pediatr Neurol 2008 Dec;15(4):216-20. doi: 10.1016/j.spen.2008.10.014. PMID: 19073331
Le Ber I, Brice A, Dürr A
Curr Neurol Neurosci Rep 2005 Sep;5(5):411-7. doi: 10.1007/s11910-005-0066-4. PMID: 16131425
Taylor AM
Best Pract Res Clin Haematol 2001 Sep;14(3):631-44. doi: 10.1053/beha.2001.0158. PMID: 11640873

Diagnosis

Worm C, Schambye MER, Mkrtchyan GV, Veviorskiy A, Shneyderman A, Ozerov IV, Zhavoronkov A, Bakula D, Scheibye-Knudsen M
Aging (Albany NY) 2024 Feb 9;16(3):2026-2046. doi: 10.18632/aging.205537. PMID: 38345566Free PMC Article
Magrino J, Munford V, Martins DJ, Homma TK, Page B, Gaubitz C, Freire BL, Lerario AM, Vilar JB, Amorin A, Leão EKE, Kok F, Menck CF, Jorge AA, Kelch BA
J Biol Chem 2023 May;299(5):104656. Epub 2023 Mar 27 doi: 10.1016/j.jbc.2023.104656. PMID: 36990216Free PMC Article
Liu W, Narayanan V
Semin Pediatr Neurol 2008 Dec;15(4):216-20. doi: 10.1016/j.spen.2008.10.014. PMID: 19073331
Taylor AM, Groom A, Byrd PJ
DNA Repair (Amst) 2004 Aug-Sep;3(8-9):1219-25. doi: 10.1016/j.dnarep.2004.04.009. PMID: 15279810
Taylor AM
Best Pract Res Clin Haematol 2001 Sep;14(3):631-44. doi: 10.1053/beha.2001.0158. PMID: 11640873

Therapy

Uchisaka N, Takahashi N, Sato M, Kikuchi A, Mochizuki S, Imai K, Nonoyama S, Ohara O, Watanabe F, Mizutani S, Hanada R, Morio T
J Pediatr 2009 Sep;155(3):435-8. doi: 10.1016/j.jpeds.2009.02.037. PMID: 19732584

Prognosis

Tarapara B, Shah F
J Biomol Struct Dyn 2023 Apr;41(6):2160-2174. Epub 2022 Jan 20 doi: 10.1080/07391102.2022.2028678. PMID: 35048780
Regal JA, Festerling TA, Buis JM, Ferguson DO
Hum Mol Genet 2013 Dec 20;22(25):5146-59. Epub 2013 Aug 2 doi: 10.1093/hmg/ddt368. PMID: 23912341Free PMC Article
Oba D, Hayashi M, Minamitani M, Hamano S, Uchisaka N, Kikuchi A, Kishimoto H, Takagi M, Morio T, Mizutani S
Acta Neuropathol 2010 Apr;119(4):513-20. Epub 2010 Jan 20 doi: 10.1007/s00401-010-0639-4. PMID: 20087742
Uchisaka N, Takahashi N, Sato M, Kikuchi A, Mochizuki S, Imai K, Nonoyama S, Ohara O, Watanabe F, Mizutani S, Hanada R, Morio T
J Pediatr 2009 Sep;155(3):435-8. doi: 10.1016/j.jpeds.2009.02.037. PMID: 19732584
Theunissen JW, Kaplan MI, Hunt PA, Williams BR, Ferguson DO, Alt FW, Petrini JH
Mol Cell 2003 Dec;12(6):1511-23. doi: 10.1016/s1097-2765(03)00455-6. PMID: 14690604

Clinical prediction guides

Fiévet A, Bellanger D, Valence S, Mobuchon L, Afenjar A, Giuliano F, Dubois d'Enghien C, Parfait B, Pedespan JM, Auger N, Rieunier G, Collet A, Burglen L, Stoppa-Lyonnet D, Stern MH
Hum Mutat 2019 Oct;40(10):1690-1699. Epub 2019 May 15 doi: 10.1002/humu.23773. PMID: 31033087
Unterholzner L
Immunobiology 2013 Nov;218(11):1312-21. Epub 2013 Jul 29 doi: 10.1016/j.imbio.2013.07.007. PMID: 23962476
Khan AO, Oystreck DT, Koenig M, Salih MA
J AAPOS 2008 Apr;12(2):186-9. Epub 2007 Dec 21 doi: 10.1016/j.jaapos.2007.09.016. PMID: 18083591
Gueven N, Chen P, Nakamura J, Becherel OJ, Kijas AW, Grattan-Smith P, Lavin MF
Neuroscience 2007 Apr 14;145(4):1418-25. Epub 2007 Jan 16 doi: 10.1016/j.neuroscience.2006.12.010. PMID: 17224243
Petrini JH
Curr Opin Cell Biol 2000 Jun;12(3):293-6. doi: 10.1016/s0955-0674(00)00091-0. PMID: 10801460

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