About Our Laboratory
Our research focuses on understanding the cellular and molecular basis of neurodegeneration. Currently we are studying human neurodegenerative syndromes that affect the cerebellum and basal ganglia to result in abnormalities of movement such as ataxia and parkinsonism.
We are testing the idea that neurodegeneration results from derangements in relatively few but strategic sub-cellular pathways. By identifying critical components of these pathways one could begin to not only understand the biology of neurodegeneration, but also embark on the design of novel therapeutic agents.
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.PMID:31097008
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.PMID:30649233
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.PMID:30590536