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Uninterrupted CAG repeat drives striatum-selective transcriptionopathy and nuclear pathogenesis in human Huntingtin BAC mice X Gu, J Richman, P Langfelder, N Wang, S Zhang, M Bañez-Coronel, ... Neuron 110 (7), 1173-1192. e7, 2022 | 34 | 2022 |
Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy YJ Kim, T Nomakuchi, F Papaleonidopoulou, L Yang, Q Zhang, ... Nature communications 13 (1), 2978, 2022 | 28 | 2022 |
Antisense oligonucleotide therapy for H3. 3K27M diffuse midline glioma Q Zhang, L Yang, YH Liu, JE Wilkinson, AR Krainer Science translational medicine 15 (691), eadd8280, 2023 | 13 | 2023 |