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Leber hereditary optic neuropathy, autosomal recessive(MC1DN38; LHONAR1)

MedGen UID:
1786310
Concept ID:
C5543589
Disease or Syndrome
Synonyms: LEBER-LIKE HEREDITARY OPTIC NEUROPATHY, AUTOSOMAL RECESSIVE 1; LHONAR; LHONAR1; MC1DN38; MITOCHONDRIAL COMPLEX I DEFICIENCY, NUCLEAR TYPE 38
 
Related genes: DNAJC30, NDUFS2
 
Monarch Initiative: MONDO:0030309
OMIM®: 618202; 619382
OMIM® Phenotypic series: PS619382

Clinical features

From HPO
Central scotoma
MedGen UID:
57750
Concept ID:
C0152191
Finding
An area of depressed vision located at the point of fixation and that interferes with central vision.
Retinal telangiectasia
MedGen UID:
57598
Concept ID:
C0154835
Disease or Syndrome
Dilatation of small blood vessels of the retina.
Reduced visual acuity
MedGen UID:
65889
Concept ID:
C0234632
Finding
Diminished clarity of vision.
Central retinal vessel vascular tortuosity
MedGen UID:
867211
Concept ID:
C4021569
Finding
The presence of an increased number of twists and turns of retinal blood vessels (arteries, arterioles, veins, venules).
Retinal nerve fiber edema
MedGen UID:
1689646
Concept ID:
C5209223
Pathologic Function
Swelling (edema) of the retinal nerve fibers.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  

Recent clinical studies

Etiology

Skorczyk-Werner A, Tońska K, Maciejczuk A, Nowomiejska K, Korwin M, Ołdak M, Wawrocka A, Krawczyński MR
Int J Mol Sci 2023 Dec 15;24(24) doi: 10.3390/ijms242417496. PMID: 38139324Free PMC Article
Rocatcher A, Desquiret-Dumas V, Charif M, Ferré M, Gohier P, Mirebeau-Prunier D, Verny C, Milea D, Lenaers G; HON Collaborators Group, Bonneau D, Reynier P, Amati-Bonneau P
Brain 2023 Feb 13;146(2):455-460. doi: 10.1093/brain/awac395. PMID: 36317462
Klopstock T, Priglinger C, Yilmaz A, Kornblum C, Distelmaier F, Prokisch H
Dtsch Arztebl Int 2021 Nov 5;118(44):741-748. doi: 10.3238/arztebl.m2021.0251. PMID: 34158150Free PMC Article
Tranchant C, Anheim M
Rev Neurol (Paris) 2016 Aug-Sep;172(8-9):524-529. Epub 2016 Jul 28 doi: 10.1016/j.neurol.2016.07.003. PMID: 27476418
Neuhann T, Rautenstrauss B
Expert Rev Neurother 2013 Apr;13(4):357-67. doi: 10.1586/ern.13.19. PMID: 23545052

Diagnosis

Lenaers G, Beaulieu C, Charif M, Gerber S, Kaplan J, Rozet JM
Brain 2023 Aug 1;146(8):3156-3161. doi: 10.1093/brain/awad131. PMID: 37071596Free PMC Article
Carelli V, La Morgia C, Yu-Wai-Man P
Handb Clin Neurol 2023;194:23-42. doi: 10.1016/B978-0-12-821751-1.00010-5. PMID: 36813316
Klopstock T, Priglinger C, Yilmaz A, Kornblum C, Distelmaier F, Prokisch H
Dtsch Arztebl Int 2021 Nov 5;118(44):741-748. doi: 10.3238/arztebl.m2021.0251. PMID: 34158150Free PMC Article
Stenton SL, Sheremet NL, Catarino CB, Andreeva NA, Assouline Z, Barboni P, Barel O, Berutti R, Bychkov I, Caporali L, Capristo M, Carbonelli M, Cascavilla ML, Charbel Issa P, Freisinger P, Gerber S, Ghezzi D, Graf E, Heidler J, Hempel M, Heon E, Itkis YS, Javasky E, Kaplan J, Kopajtich R, Kornblum C, Kovacs-Nagy R, Krylova TD, Kunz WS, La Morgia C, Lamperti C, Ludwig C, Malacarne PF, Maresca A, Mayr JA, Meisterknecht J, Nevinitsyna TA, Palombo F, Pode-Shakked B, Shmelkova MS, Strom TM, Tagliavini F, Tzadok M, van der Ven AT, Vignal-Clermont C, Wagner M, Zakharova EY, Zhorzholadze NV, Rozet JM, Carelli V, Tsygankova PG, Klopstock T, Wittig I, Prokisch H
J Clin Invest 2021 Mar 15;131(6) doi: 10.1172/JCI138267. PMID: 33465056Free PMC Article
Zeviani M, Tiranti V, Piantadosi C
Medicine (Baltimore) 1998 Jan;77(1):59-72. doi: 10.1097/00005792-199801000-00006. PMID: 9465864

Therapy

Hu JL, Hsu CC, Hsiao YJ, Lin YY, Lai WY, Liu YH, Wang CL, Ko YL, Tsai ML, Tseng HC, Chien Y, Yang YP
J Chin Med Assoc 2024 Jan 1;87(1):12-16. Epub 2023 Nov 28 doi: 10.1097/JCMA.0000000000001031. PMID: 38016117

Prognosis

Hotta Y, Torii K, Takayama M
Jpn J Ophthalmol 2024 Sep;68(5):401-418. Epub 2024 Sep 14 doi: 10.1007/s10384-024-01109-8. PMID: 39271608Free PMC Article
Roomets E, Mauring L
Eur J Ophthalmol 2024 Sep;34(5):NP1-NP7. Epub 2024 May 7 doi: 10.1177/11206721241254408. PMID: 38715355Free PMC Article
Zou XH, Guo XX, Su HZ, Wang C, Dong EL, Wang N, Chen WJ, Zhang QJ
J Mol Neurosci 2019 Aug;68(4):640-646. Epub 2019 May 10 doi: 10.1007/s12031-019-01319-7. PMID: 31077085
Lenaers G, Hamel C, Delettre C, Amati-Bonneau P, Procaccio V, Bonneau D, Reynier P, Milea D
Orphanet J Rare Dis 2012 Jul 9;7:46. doi: 10.1186/1750-1172-7-46. PMID: 22776096Free PMC Article
Finsterer J
Acta Haematol 2007;118(2):88-98. Epub 2007 Jul 18 doi: 10.1159/000105676. PMID: 17637511

Clinical prediction guides

Hotta Y, Torii K, Takayama M
Jpn J Ophthalmol 2024 Sep;68(5):401-418. Epub 2024 Sep 14 doi: 10.1007/s10384-024-01109-8. PMID: 39271608Free PMC Article
Gupta PR, Gospe SM , III
Ophthalmic Genet 2023 Oct;44(5):469-474. Epub 2022 Oct 19 doi: 10.1080/13816810.2022.2135112. PMID: 36262091
Neuhann T, Rautenstrauss B
Expert Rev Neurother 2013 Apr;13(4):357-67. doi: 10.1586/ern.13.19. PMID: 23545052
Qu J, Wang Y, Tong Y, Zhou X, Zhao F, Yang L, Zhang S, Zhang J, West CE, Guan MX
Invest Ophthalmol Vis Sci 2010 Oct;51(10):4906-12. Epub 2010 Apr 30 doi: 10.1167/iovs.09-5027. PMID: 20435583Free PMC Article
Richard M, Roepman R, Aartsen WM, van Rossum AG, den Hollander AI, Knust E, Wijnholds J, Cremers FP
Hum Mol Genet 2006 Oct 15;15 Spec No 2:R235-43. doi: 10.1093/hmg/ddl195. PMID: 16987889

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