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Mitochondrial DNA depletion syndrome 12

MedGen UID:
1377952
Concept ID:
C4321247
Disease or Syndrome
Synonym: Mitochondrial DNA Depletion Syndrome 12
 
Related gene: SLC25A4

Definition

An inherited condition caused by mutation(s) in the SLC25A4 gene, encoding ADP/ATP translocase 1. It is characterized by hypertrophic cardiomyopathy. [from NCI]

Professional guidelines

PubMed

Domínguez-González C, Madruga-Garrido M, Hirano M, Martí I, Martín MA, Munell F, Nascimento A, Olivé M, Quan J, Sardina MD, Martí R, Paradas C
Orphanet J Rare Dis 2021 Oct 2;16(1):407. doi: 10.1186/s13023-021-02030-w. PMID: 34600563Free PMC Article
Carrozzo R, Verrigni D, Rasmussen M, de Coo R, Amartino H, Bianchi M, Buhas D, Mesli S, Naess K, Born AP, Woldseth B, Prontera P, Batbayli M, Ravn K, Joensen F, Cordelli DM, Santorelli FM, Tulinius M, Darin N, Duno M, Jouvencel P, Burlina A, Stangoni G, Bertini E, Redonnet-Vernhet I, Wibrand F, Dionisi-Vici C, Uusimaa J, Vieira P, Osorio AN, McFarland R, Taylor RW, Holme E, Ostergaard E
J Inherit Metab Dis 2016 Mar;39(2):243-52. Epub 2015 Oct 16 doi: 10.1007/s10545-015-9894-9. PMID: 26475597

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
Vara R, Pinon M, Fratter C, Hegarty R, Hadzic N
J Inherit Metab Dis 2023 Jul;46(4):634-648. Epub 2023 May 28 doi: 10.1002/jimd.12633. PMID: 37204315
Longchamps RJ, Yang SY, Castellani CA, Shi W, Lane J, Grove ML, Bartz TM, Sarnowski C, Liu C, Burrows K, Guyatt AL, Gaunt TR, Kacprowski T, Yang J, De Jager PL, Yu L, Bergman A, Xia R, Fornage M, Feitosa MF, Wojczynski MK, Kraja AT, Province MA, Amin N, Rivadeneira F, Tiemeier H, Uitterlinden AG, Broer L, Van Meurs JBJ, Van Duijn CM, Raffield LM, Lange L, Rich SS, Lemaitre RN, Goodarzi MO, Sitlani CM, Mak ACY, Bennett DA, Rodriguez S, Murabito JM, Lunetta KL, Sotoodehnia N, Atzmon G, Ye K, Barzilai N, Brody JA, Psaty BM, Taylor KD, Rotter JI, Boerwinkle E, Pankratz N, Arking DE
Hum Genet 2022 Jan;141(1):127-146. Epub 2021 Dec 2 doi: 10.1007/s00439-021-02394-w. PMID: 34859289Free PMC Article
Oller J, Gabandé-Rodríguez E, Ruiz-Rodríguez MJ, Desdín-Micó G, Aranda JF, Rodrigues-Diez R, Ballesteros-Martínez C, Blanco EM, Roldan-Montero R, Acuña P, Forteza Gil A, Martín-López CE, Nistal JF, Lino Cardenas CL, Lindsay ME, Martín-Ventura JL, Briones AM, Redondo JM, Mittelbrunn M
Circulation 2021 May 25;143(21):2091-2109. Epub 2021 Mar 12 doi: 10.1161/CIRCULATIONAHA.120.051171. PMID: 33709773Free PMC Article
Alberio S, Mineri R, Tiranti V, Zeviani M
Mitochondrion 2007 Feb-Apr;7(1-2):6-12. Epub 2006 Dec 5 doi: 10.1016/j.mito.2006.11.010. PMID: 17280874

Diagnosis

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
Domínguez-González C, Madruga-Garrido M, Hirano M, Martí I, Martín MA, Munell F, Nascimento A, Olivé M, Quan J, Sardina MD, Martí R, Paradas C
Orphanet J Rare Dis 2021 Oct 2;16(1):407. doi: 10.1186/s13023-021-02030-w. PMID: 34600563Free PMC Article
Ramón J, Vila-Julià F, Molina-Granada D, Molina-Berenguer M, Melià MJ, García-Arumí E, Torres-Torronteras J, Cámara Y, Martí R
Int J Mol Sci 2021 Jun 16;22(12) doi: 10.3390/ijms22126447. PMID: 34208592Free PMC Article
Shimura M, Kuranobu N, Ogawa-Tominaga M, Akiyama N, Sugiyama Y, Ebihara T, Fushimi T, Ichimoto K, Matsunaga A, Tsuruoka T, Kishita Y, Umetsu S, Inui A, Fujisawa T, Tanikawa K, Ito R, Fukuda A, Murakami J, Kaji S, Kasahara M, Shiraki K, Ohtake A, Okazaki Y, Murayama K
Orphanet J Rare Dis 2020 Jul 24;15(1):169. doi: 10.1186/s13023-020-01441-5. PMID: 32703289Free PMC Article
Poulton J, Holt IJ
Neuromuscul Disord 2009 Jun;19(6):439-43. Epub 2009 May 21 doi: 10.1016/j.nmd.2009.04.009. PMID: 19464176

Therapy

Bermejo-Guerrero L, Hernández-Voth A, Serrano-Lorenzo P, Blázquez A, Martin-Jimenez P, Martin MA, Domínguez-González C
Mitochondrion 2024 May;76:101879. Epub 2024 Apr 9 doi: 10.1016/j.mito.2024.101879. PMID: 38599303
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
Gordon N
Dev Med Child Neurol 2006 Dec;48(12):1001-3. doi: 10.1017/S0012162206002209. PMID: 17109792
Hoy JF, Gahan ME, Carr A, Smith D, Lewin SR, Wesselingh S, Cooper DA
J Infect Dis 2004 Aug 15;190(4):688-92. Epub 2004 Jul 12 doi: 10.1086/422602. PMID: 15272394
Nolan D, Hammond E, Martin A, Taylor L, Herrmann S, McKinnon E, Metcalf C, Latham B, Mallal S
AIDS 2003 Jun 13;17(9):1329-38. doi: 10.1097/00002030-200306130-00007. PMID: 12799554

Prognosis

Keshavan N, Rahman S
Mol Genet Metab 2024 Sep-Oct;143(1-2):108554. Epub 2024 Jul 24 doi: 10.1016/j.ymgme.2024.108554. PMID: 39079226
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
Domínguez-González C, Madruga-Garrido M, Hirano M, Martí I, Martín MA, Munell F, Nascimento A, Olivé M, Quan J, Sardina MD, Martí R, Paradas C
Orphanet J Rare Dis 2021 Oct 2;16(1):407. doi: 10.1186/s13023-021-02030-w. PMID: 34600563Free PMC Article
Shimura M, Kuranobu N, Ogawa-Tominaga M, Akiyama N, Sugiyama Y, Ebihara T, Fushimi T, Ichimoto K, Matsunaga A, Tsuruoka T, Kishita Y, Umetsu S, Inui A, Fujisawa T, Tanikawa K, Ito R, Fukuda A, Murakami J, Kaji S, Kasahara M, Shiraki K, Ohtake A, Okazaki Y, Murayama K
Orphanet J Rare Dis 2020 Jul 24;15(1):169. doi: 10.1186/s13023-020-01441-5. PMID: 32703289Free PMC Article
Domínguez-González C, Hernández-Laín A, Rivas E, Hernández-Voth A, Sayas Catalán J, Fernández-Torrón R, Fuiza-Luces C, García García J, Morís G, Olivé M, Miralles F, Díaz-Manera J, Caballero C, Méndez-Ferrer B, Martí R, García Arumi E, Badosa MC, Esteban J, Jimenez-Mallebrera C, Encinar AB, Arenas J, Hirano M, Martin MÁ, Paradas C
Orphanet J Rare Dis 2019 May 6;14(1):100. doi: 10.1186/s13023-019-1071-z. PMID: 31060578Free PMC Article

Clinical prediction guides

Keshavan N, Rahman S
Mol Genet Metab 2024 Sep-Oct;143(1-2):108554. Epub 2024 Jul 24 doi: 10.1016/j.ymgme.2024.108554. PMID: 39079226
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
Key J, Gispert S, Koornneef L, Sleddens-Linkels E, Kohli A, Torres-Odio S, Koepf G, Amr S, Reichlmeir M, Harter PN, West AP, Münch C, Baarends WM, Auburger G
Cells 2022 Dec 22;12(1) doi: 10.3390/cells12010052. PMID: 36611846Free PMC Article
Kodani A, Yamaguchi M, Itoh R, Huynh MA, Yoshida H
Sci Rep 2022 Dec 31;12(1):22632. doi: 10.1038/s41598-022-27329-x. PMID: 36587049Free PMC Article

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