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Page 1
Allele-selective lowering of mutant HTT protein by HTT-LC3 linker compounds.
Li Z, Wang C, Wang Z, Zhu C, Li J, Sha T, Ma L, Gao C, Yang Y, Sun Y, Wang J, Sun X, Lu C, Difiglia M, Mei Y, Ding C, Luo S, Dang Y, Ding Y, Fei Y, Lu B. Li Z, et al. Nature. 2019 Nov;575(7781):203-209. doi: 10.1038/s41586-019-1722-1. Epub 2019 Oct 30. Nature. 2019. PMID: 31666698
A Robust Assay to Monitor Ataxin-3 Amyloid Fibril Assembly.
Figueiredo F, Lopes-Marques M, Almeida B, Matscheko N, Martins PM, Silva A, Macedo-Ribeiro S. Figueiredo F, et al. Cells. 2022 Jun 19;11(12):1969. doi: 10.3390/cells11121969. Cells. 2022. PMID: 35741099 Free PMC article.
Polyglutamine-Expanded Ataxin-3: A Target Engagement Marker for Spinocerebellar Ataxia Type 3 in Peripheral Blood.
Hübener-Schmid J, Kuhlbrodt K, Peladan J, Faber J, Santana MM, Hengel H, Jacobi H, Reetz K, Garcia-Moreno H, Raposo M, van Gaalen J, Infante J, Steiner KM, de Vries J, Verbeek MM, Giunti P, Pereira de Almeida L, Lima M, van de Warrenburg B, Schöls L, Klockgether T, Synofzik M; European Spinocerebellar Ataxia Type-3/Machado-Joseph Disease Initiative (ESMI) Study Group; Riess O. Hübener-Schmid J, et al. Mov Disord. 2021 Nov;36(11):2675-2681. doi: 10.1002/mds.28749. Epub 2021 Aug 16. Mov Disord. 2021. PMID: 34397117
784 results