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Identification and Optimization of RNA-Splicing Modulators as Huntingtin Protein-Lowering Agents for the Treatment of Huntington’s Disease2023 Journal of Medicinal Chemistry article Neuroscience Genetic Neurodegenerative Diseases Open access cited by 1 patent
Longbin Liu, Karine Malagu, Alan F. Haughan, Vinod K. Khetarpal, Andrew J. Stott, William Esmieu, +41 more
8citations -
Influence of ATXN2 intermediate CAG repeats, 9bp duplication and alternative splicing on SCA3 pathogenesis2025 Acta Neuropathologica Communications article Neuroscience Genetic Neurodegenerative Diseases Open access
Marilena Lauerer, Jennifer Faber, Nicolas Casadei, Magda M. Santana, Georg W. J. Auburger, Michaela Pogoda, +22 more
3citations
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