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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

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

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
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
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
Hirano M, Berardo A, Barca E, Emmanuele V, Quinzii C, Simpson CV, Engelstad K, Rosales XQ, Thompson JLP
J Inherit Metab Dis 2021 Mar;44(2):292-300. Epub 2021 Jan 4 doi: 10.1002/jimd.12353. PMID: 33368420Free PMC Article
Wang L, Limongelli A, Vila MR, Carrara F, Zeviani M, Eriksson S
Mol Genet Metab 2005 Jan;84(1):75-82. doi: 10.1016/j.ymgme.2004.09.005. PMID: 15639197

Diagnosis

Uchida H, Sakamoto S, Shimizu S, Yanagi Y, Fukuda A, Horikawa R, Ito R, Matsunaga A, Murayama K, Kasahara M
Pediatr Transplant 2021 Dec;25(8):e14091. Epub 2021 Jul 15 doi: 10.1111/petr.14091. PMID: 34265160
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
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
Nguyen KV, Sharief FS, Chan SS, Copeland WC, Naviaux RK
J Hepatol 2006 Jul;45(1):108-16. Epub 2006 Feb 20 doi: 10.1016/j.jhep.2005.12.026. PMID: 16545482

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
Vita G, Vita GL, Musumeci O, Rodolico C, Messina S
Neurol Sci 2019 Apr;40(4):671-681. Epub 2019 Feb 25 doi: 10.1007/s10072-019-03764-z. PMID: 30805745
Hirano M, Emmanuele V, Quinzii CM
Essays Biochem 2018 Jul 20;62(3):467-481. doi: 10.1042/EBC20170114. PMID: 29980632Free PMC Article
Saito K, Kimura N, Oda N, Shimomura H, Kumada T, Miyajima T, Murayama K, Tanaka M, Fujii T
Biochim Biophys Acta 2012 May;1820(5):632-6. Epub 2011 Aug 11 doi: 10.1016/j.bbagen.2011.08.006. PMID: 21855607

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
Hirano M, Berardo A, Barca E, Emmanuele V, Quinzii C, Simpson CV, Engelstad K, Rosales XQ, Thompson JLP
J Inherit Metab Dis 2021 Mar;44(2):292-300. Epub 2021 Jan 4 doi: 10.1002/jimd.12353. PMID: 33368420Free PMC Article
Keshavan N, Abdenur J, Anderson G, Assouline Z, Barcia G, Bouhikbar L, Chakrapani A, Cleary M, Cohen MC, Feillet F, Fratter C, Hauser N, Jacques T, Lam A, McCullagh H, Phadke R, Rötig A, Sharrard M, Simon M, Smith C, Sommerville EW, Taylor RW, Yue WW, Rahman S
Genet Med 2020 Jan;22(1):199-209. Epub 2019 Aug 29 doi: 10.1038/s41436-019-0613-z. PMID: 31462754
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 L, Limongelli A, Vila MR, Carrara F, Zeviani M, Eriksson S
Mol Genet Metab 2005 Jan;84(1):75-82. doi: 10.1016/j.ymgme.2004.09.005. PMID: 15639197

Clinical prediction guides

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
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
Jou C, Nascimento A, Codina A, Montoya J, López-Gallardo E, Emperador S, Ruiz-Pesini E, Montero R, Natera-de Benito D, Ortez CI, Marquez J, Zelaya MV, Gutierrez-Mata A, Badosa C, Carrera-García L, Expósito-Escudero J, Roldán M, Camara Y, Marti R, Ferrer I, Jimenez-Mallebrera C, Artuch R
Int J Mol Sci 2022 Sep 20;23(19) doi: 10.3390/ijms231911002. PMID: 36232299Free PMC Article
Hirano M, Berardo A, Barca E, Emmanuele V, Quinzii C, Simpson CV, Engelstad K, Rosales XQ, Thompson JLP
J Inherit Metab Dis 2021 Mar;44(2):292-300. Epub 2021 Jan 4 doi: 10.1002/jimd.12353. PMID: 33368420Free PMC Article

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