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The role of neurofilament aggregation in neurodegeneration: lessons from rare inherited neurological disorders.

Didonna A, Opal P.

Mol Neurodegener. 2019 May 16;14(1):19. doi: 10.1186/s13024-019-0318-4. Review.


Self-assembling vascular endothelial growth factor nanoparticles improve function in spinocerebellar ataxia type 1.

Hu YS, Do J, Edamakanti CR, Kini AR, Martina M, Stupp SI, Opal P.

Brain. 2019 Feb 1;142(2):312-321. doi: 10.1093/brain/awy328.


Loss of the dystonia gene Thap1 leads to transcriptional deficits that converge on common pathogenic pathways in dystonic syndromes.

Frederick NM, Shah PV, Didonna A, Langley MR, Kanthasamy AG, Opal P.

Hum Mol Genet. 2019 Apr 15;28(8):1343-1356. doi: 10.1093/hmg/ddy433.


Trinucleotide Repeat Expansion Diseases, RNAi, and Cancer.

Murmann AE, Yu J, Opal P, Peter ME.

Trends Cancer. 2018 Oct;4(10):684-700. doi: 10.1016/j.trecan.2018.08.004. Epub 2018 Sep 26. Review.


Mutant ataxin1 disrupts cerebellar development in spinocerebellar ataxia type 1.

Edamakanti CR, Do J, Didonna A, Martina M, Opal P.

J Clin Invest. 2018 Jun 1;128(6):2252-2265. doi: 10.1172/JCI96765. Epub 2018 Apr 23.


ANP32A regulates histone H3 acetylation and promotes leukemogenesis.

Yang X, Lu B, Sun X, Han C, Fu C, Xu K, Wang M, Li D, Chen Z, Opal P, Wen Q, Crispino JD, Wang QF, Huang Z.

Leukemia. 2018 Jul;32(7):1587-1597. doi: 10.1038/s41375-018-0010-7. Epub 2018 Feb 2.


ANP32A dysregulation contributes to abnormal megakaryopoiesis in acute megakaryoblastic leukemia.

Sun X, Lu B, Han C, Qiu W, Jin Q, Li D, Li Q, Yang Q, Wen Q, Opal P, Kini AR, Crispino JD, Huang Z.

Blood Cancer J. 2017 Dec 22;7(12):661. doi: 10.1038/s41408-017-0031-x. No abstract available.


Spinocerebellar Ataxia Type 1.

Opal P, Ashizawa T.

In: Adam MP, Ardinger HH, Pagon RA, Wallace SE, Bean LJH, Stephens K, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019.
1998 Oct 1 [updated 2017 Jun 22].


The role of gigaxonin in the degradation of the glial-specific intermediate filament protein GFAP.

Lin NH, Huang YS, Opal P, Goldman RD, Messing A, Perng MD.

Mol Biol Cell. 2016 Dec 15;27(25):3980-3990. Epub 2016 Oct 26.


Intermediate filament aggregates cause mitochondrial dysmotility and increase energy demands in giant axonal neuropathy.

Israeli E, Dryanovski DI, Schumacker PT, Chandel NS, Singer JD, Julien JP, Goldman RD, Opal P.

Hum Mol Genet. 2016 Jun 1;25(11):2143-2157. Epub 2016 Mar 21.


Advances in Sequencing Technologies for Understanding Hereditary Ataxias: A Review.

Didonna A, Opal P.

JAMA Neurol. 2016 Dec 1;73(12):1485-1490. doi: 10.1001/jamaneurol.2016.3097. Review.


Paraneoplastic cerebellar degeneration with anti-Yo antibodies - a review.

Venkatraman A, Opal P.

Ann Clin Transl Neurol. 2016 Jun 30;3(8):655-63. doi: 10.1002/acn3.328. eCollection 2016 Aug. Review.


Mutant Ataxin-1 Inhibits Neural Progenitor Cell Proliferation in SCA1.

Cvetanovic M, Hu YS, Opal P.

Cerebellum. 2017 Apr;16(2):340-347. doi: 10.1007/s12311-016-0794-9.


Abnormal intermediate filament organization alters mitochondrial motility in giant axonal neuropathy fibroblasts.

Lowery J, Jain N, Kuczmarski ER, Mahammad S, Goldman A, Gelfand VI, Opal P, Goldman RD.

Mol Biol Cell. 2016 Feb 15;27(4):608-16. doi: 10.1091/mbc.E15-09-0627. Epub 2015 Dec 23.


Neuronal Atrophy Early in Degenerative Ataxia Is a Compensatory Mechanism to Regulate Membrane Excitability.

Dell'Orco JM, Wasserman AH, Chopra R, Ingram MA, Hu YS, Singh V, Wulff H, Opal P, Orr HT, Shakkottai VG.

J Neurosci. 2015 Aug 12;35(32):11292-307. doi: 10.1523/JNEUROSCI.1357-15.2015.


The promise and perils of HDAC inhibitors in neurodegeneration.

Didonna A, Opal P.

Ann Clin Transl Neurol. 2015 Jan;2(1):79-101. doi: 10.1002/acn3.147. Epub 2014 Dec 3. Review.


Early activation of microglia and astrocytes in mouse models of spinocerebellar ataxia type 1.

Cvetanovic M, Ingram M, Orr H, Opal P.

Neuroscience. 2015 Mar 19;289:289-99. doi: 10.1016/j.neuroscience.2015.01.003. Epub 2015 Jan 14.


An investigation of diffusion imaging techniques in the evaluation of spinocerebellar ataxia and multisystem atrophy.

Rozenfeld MN, Nemeth AJ, Walker MT, Mohan P, Wang X, Parrish TB, Opal P.

J Clin Neurosci. 2015 Jan;22(1):166-72. doi: 10.1016/j.jocn.2014.08.006. Epub 2014 Nov 26.


The histone deacetylase HDAC3 is essential for Purkinje cell function, potentially complicating the use of HDAC inhibitors in SCA1.

Venkatraman A, Hu YS, Didonna A, Cvetanovic M, Krbanjevic A, Bilesimo P, Opal P.

Hum Mol Genet. 2014 Jul 15;23(14):3733-45. doi: 10.1093/hmg/ddu081. Epub 2014 Mar 4.


Giant axonal neuropathy-associated gigaxonin mutations impair intermediate filament protein degradation.

Mahammad S, Murthy SN, Didonna A, Grin B, Israeli E, Perrot R, Bomont P, Julien JP, Kuczmarski E, Opal P, Goldman RD.

J Clin Invest. 2013 May;123(5):1964-75. doi: 10.1172/JCI66387. Epub 2013 Apr 15.

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