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Mitochondrial DNA depletion syndrome

MedGen UID:
452449
Concept ID:
C0342782
Disease or Syndrome
Synonym: mitochondrial DNA depletion
SNOMED CT: Depletion of mitochondrial deoxyribonucleic acid (237995002); Depletion of mitochondrial DNA (237995002)
 
Monarch Initiative: MONDO:0018158
OMIM® Phenotypic series: PS603041
Orphanet: ORPHA35698

Definition

A clinically heterogeneous group of mitochondrial disorders characterized by a reduction of the mitochondrial DNA copy number in affected tissues without mutations or rearrangements in the mitochondrial DNA. It is phenotypically heterogeneous, and can affect a specific organ or a combination of organs, with the main presentations described being either hepatocerebral (i.e. hepatic dysfunction, psychomotor delay), myopathic (i.e. hypotonia, muscle weakness, bulbar weakness), encephalomyopathic (i.e. hypotonia, muscle weakness, psychomotor delay) or neurogastrointestinal (i.e gastrointestinal dysmotility, peripheral neuropathy). Additional phenotypes include fatal infantile lactic acidosis with methylmalonic aciduria, spastic ataxia (early-onset spastic ataxia-neuropathy syndrome), and Alpers syndrome. [from ORDO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVMitochondrial DNA depletion syndrome
Follow this link to review classifications for Mitochondrial DNA depletion syndrome in Orphanet.

Professional guidelines

PubMed

Han X, Li H, Deng J, Zhuo X, Liu Z, Xu M, Feng W, Chen S, Fang F
Neurol Sci 2024 Nov;45(11):5465-5480. Epub 2024 Jun 4 doi: 10.1007/s10072-024-07586-6. PMID: 38831166
Amtmann D, Gammaitoni AR, Galer BS, Salem R, Jensen MP
Mitochondrion 2023 Jan;68:1-9. Epub 2022 Oct 29 doi: 10.1016/j.mito.2022.10.003. PMID: 36374792

Recent clinical studies

Etiology

Chen Y, Jiao D, Liu Y, Xu X, Wang Y, Luo X, Saiyin H, Li Y, Gao K, Chen Y, Zhao SM, Ma L, Wang C
Cell Death Differ 2023 Oct;30(10):2351-2363. Epub 2023 Aug 11 doi: 10.1038/s41418-023-01205-1. PMID: 37568009Free PMC Article
Guo J, Duan L, He X, Li S, Wu Y, Xiang G, Bao F, Yang L, Shi H, Gao M, Zheng L, Hu H, Liu X
Adv Sci (Weinh) 2021 May;8(10):2004680. Epub 2021 Mar 8 doi: 10.1002/advs.202004680. PMID: 34026460Free PMC Article
Montero R, Grazina M, López-Gallardo E, Montoya J, Briones P, Navarro-Sastre A, Land JM, Hargreaves IP, Artuch R; Coenzyme Q₁₀ Deficiency Study Group
Mitochondrion 2013 Jul;13(4):337-41. Epub 2013 Apr 11 doi: 10.1016/j.mito.2013.04.001. PMID: 23583954
Kasapkara CS, Tümer L, Küçükçongar A, Hasanoğlu A, Seneca S, De Meirleir L
J Pediatr Gastroenterol Nutr 2013 Nov;57(5):e28-9. doi: 10.1097/MPG.0b013e31826bd4ed. PMID: 22868686
Rabinowitz SS, Gelfond D, Chen CK, Gloster ES, Whitington PF, Sacconi S, Salviati L, DiMauro S
J Pediatr Gastroenterol Nutr 2004 Feb;38(2):216-20. doi: 10.1097/00005176-200402000-00022. PMID: 14734888

Diagnosis

Guo J, Duan L, He X, Li S, Wu Y, Xiang G, Bao F, Yang L, Shi H, Gao M, Zheng L, Hu H, Liu X
Adv Sci (Weinh) 2021 May;8(10):2004680. Epub 2021 Mar 8 doi: 10.1002/advs.202004680. PMID: 34026460Free PMC Article
de Barcelos IP, Emmanuele V, Hirano M
Curr Opin Neurol 2019 Oct;32(5):715-721. doi: 10.1097/WCO.0000000000000743. PMID: 31408013Free PMC Article
Montero R, Grazina M, López-Gallardo E, Montoya J, Briones P, Navarro-Sastre A, Land JM, Hargreaves IP, Artuch R; Coenzyme Q₁₀ Deficiency Study Group
Mitochondrion 2013 Jul;13(4):337-41. Epub 2013 Apr 11 doi: 10.1016/j.mito.2013.04.001. PMID: 23583954
Kasapkara CS, Tümer L, Küçükçongar A, Hasanoğlu A, Seneca S, De Meirleir L
J Pediatr Gastroenterol Nutr 2013 Nov;57(5):e28-9. doi: 10.1097/MPG.0b013e31826bd4ed. PMID: 22868686
Rabinowitz SS, Gelfond D, Chen CK, Gloster ES, Whitington PF, Sacconi S, Salviati L, DiMauro S
J Pediatr Gastroenterol Nutr 2004 Feb;38(2):216-20. doi: 10.1097/00005176-200402000-00022. PMID: 14734888

Therapy

Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ, Goldstein A, Gorman GS, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Brown DA, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators
Neurology 2023 Jul 18;101(3):e238-e252. Epub 2023 Jun 2 doi: 10.1212/WNL.0000000000207402. PMID: 37268435Free PMC Article
de Barcelos IP, Emmanuele V, Hirano M
Curr Opin Neurol 2019 Oct;32(5):715-721. doi: 10.1097/WCO.0000000000000743. PMID: 31408013Free PMC Article
Hirano M, Emmanuele V, Quinzii CM
Essays Biochem 2018 Jul 20;62(3):467-481. doi: 10.1042/EBC20170114. PMID: 29980632Free PMC Article
Cámara Y, González-Vioque E, Scarpelli M, Torres-Torronteras J, Caballero A, Hirano M, Martí R
Hum Mol Genet 2014 May 1;23(9):2459-67. Epub 2013 Dec 20 doi: 10.1093/hmg/ddt641. PMID: 24362886Free PMC Article
Priego EM, Karlsson A, Gago F, Camarasa MJ, Balzarini J, Pérez-Pérez MJ
Curr Pharm Des 2012;18(20):2981-94. doi: 10.2174/138161212800672787. PMID: 22571666

Prognosis

Samanta A, Srivastava A, Mandal K, Sarma MS, Poddar U
Indian J Gastroenterol 2023 Aug;42(4):569-574. Epub 2023 Feb 8 doi: 10.1007/s12664-022-01281-0. PMID: 36753038
Remtulla S, Emilie Nguyen CT, Prasad C, Campbell C
Pediatr Neurol 2019 Jan;90:61-65. Epub 2018 Aug 9 doi: 10.1016/j.pediatrneurol.2018.08.007. PMID: 30391088
Wang J, Kim E, Dai H, Stefans V, Vogel H, Al Jasmi F, Schrier Vergano SA, Castro D, Bernes S, Bhambhani V, Long C, El-Hattab AW, Wong LJ
Mol Genet Metab 2018 Jun;124(2):124-130. Epub 2018 Apr 28 doi: 10.1016/j.ymgme.2018.04.012. PMID: 29735374
Kasapkara CS, Tümer L, Küçükçongar A, Hasanoğlu A, Seneca S, De Meirleir L
J Pediatr Gastroenterol Nutr 2013 Nov;57(5):e28-9. doi: 10.1097/MPG.0b013e31826bd4ed. PMID: 22868686
Rabinowitz SS, Gelfond D, Chen CK, Gloster ES, Whitington PF, Sacconi S, Salviati L, DiMauro S
J Pediatr Gastroenterol Nutr 2004 Feb;38(2):216-20. doi: 10.1097/00005176-200402000-00022. PMID: 14734888

Clinical prediction guides

Hidalgo-Gutierrez A, Shintaku J, Ramon J, Barriocanal-Casado E, Pesini A, Saneto RP, Garrabou G, Milisenda JC, Matas-Garcia A, Gort L, Ugarteburu O, Gu Y, Koganti L, Wang T, Tadesse S, Meneri M, Sciacco M, Wang S, Tanji K, Horwitz MS, Dorschner MO, Mansukhani M, Comi GP, Ronchi D, Marti R, Ribes A, Tort F, Hirano M
Ann Neurol 2024 Dec;96(6):1209-1224. Epub 2024 Sep 4 doi: 10.1002/ana.27071. PMID: 39230499Free PMC Article
Gao K, Chen Y, Mo R, Wang C
Autophagy 2024 Feb;20(2):460-462. Epub 2024 Jan 25 doi: 10.1080/15548627.2023.2274260. PMID: 37876279Free PMC Article
Chen Y, Jiao D, Liu Y, Xu X, Wang Y, Luo X, Saiyin H, Li Y, Gao K, Chen Y, Zhao SM, Ma L, Wang C
Cell Death Differ 2023 Oct;30(10):2351-2363. Epub 2023 Aug 11 doi: 10.1038/s41418-023-01205-1. PMID: 37568009Free PMC Article
Karaa A, Bertini E, Carelli V, Cohen BH, Enns GM, Falk MJ, Goldstein A, Gorman GS, Haas R, Hirano M, Klopstock T, Koenig MK, Kornblum C, Lamperti C, Lehman A, Longo N, Molnar MJ, Parikh S, Phan H, Pitceathly RDS, Saneto R, Scaglia F, Servidei S, Tarnopolsky M, Toscano A, Van Hove JLK, Vissing J, Vockley J, Finman JS, Brown DA, Shiffer JA, Mancuso M; MMPOWER-3 Trial Investigators
Neurology 2023 Jul 18;101(3):e238-e252. Epub 2023 Jun 2 doi: 10.1212/WNL.0000000000207402. PMID: 37268435Free PMC Article
Samanta A, Srivastava A, Mandal K, Sarma MS, Poddar U
Indian J Gastroenterol 2023 Aug;42(4):569-574. Epub 2023 Feb 8 doi: 10.1007/s12664-022-01281-0. PMID: 36753038

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