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Cell Replacement Therapy Improves Pathological Hallmarks in a Mouse Model of Leukodystrophy Vanishing White Matter
March 5, 2019
Dooves S, Leferink PS, Krabbenborg S, Breeuwsma N, Bots S, Hillen AEJ, Jacobs G, van der Knaap MS, Heine VM.
Stem Cell Reports; 12(3):441-450. doi: 10.1016/j.stemcr.2019.01.018. Epub 2019 Feb 21. PMID: 30799272; PMCID: PMC6411482.
Bergmann glia translocation: a new disease marker for vanishing white matter identifies therapeutic effects of Guanabenz treatment
June 2018
Dooves S, Bugiani M, Wisse LE, Abbink TEM, van der Knaap MS, Heine VM.
Neuropathol Appl Neurobiol; 44(4):391-403. doi: 10.1111/nan.12411. Epub 2017 Aug 1. PMID: 28953319.
eIF2B activator prevents neurological defects caused by a chronic integrated stress response
January 9, 2019
Wong YL, LeBon L, Basso AM, Kohlhaas KL, Nikkel AL, Robb HM, Donnelly-Roberts DL, Prakash J, Swensen AM, Rubinstein ND, Krishnan S, McAllister FE, Haste NV, O'Brien JJ, Roy M, Ireland A, Frost JM, Shi L, Riedmaier S, Martin K, Dart MJ, Sidrauski C.
8:e42940. doi: 10.7554/eLife.42940. PMID: 30624206; PMCID: PMC6326728.
The small molecule ISRIB rescues the stability and activity of Vanishing White Matter Disease eIF2B mutant complexes
February 28, 2018
Wong YL, LeBon L, Edalji R, Lim HB, Sun C, Sidrauski C.
7:e32733. doi: 10.7554/eLife.32733. PMID: 29489452; PMCID: PMC5829914.
Drug Screening Identifies Sigma-1-Receptor as a Target for the Therapy of VWM Leukodystrophy
September 18, 2018
Atzmon A, Herrero M, Sharet-Eshed R, Gilad Y, Senderowitz H, Elroy-Stein O.
Front Mol Neurosci. 2018;11:336. Published 2018 Sep 18. doi:10.3389/fnmol.2018.00336
Vanishing white matter: deregulated integrated stress response as therapy target
June 28, 2021
Abbink TEM, Wisse LE, Jaku E, Thiecke MJ, Voltolini-González D, Fritsen H, Bobeldijk S, Ter Braak TJ, Polder E, Postma NL, Bugiani M, Struijs EA, Verheijen M, Straat N, van der Sluis S, Thomas AAM, Molenaar D, van der Knaap MS.
Ann Clin Transl Neurol. 2019 Aug;6(8):1407-1422. doi: 10.1002/acn3.50826. Epub 2019 Jul 18. PMID: 31402619; PMCID: PMC6689685.
Identification of repurposable cytoprotective drugs in vanishing white matter disease patient-derived cells
August 5, 2021
Ng, N., Cabral-da-Silva, M.C., Maksour, S. et al.
transl med commun 5, 18 (2020). https://doi.org/10.1186/s41231-020-00071-0