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Modeling key pathological features of frontotemporal dementia with C9ORF72 repeat expansion in iPSC-derived human neurons S Almeida, E Gascon, H Tran, HJ Chou, TF Gendron, S DeGroot, ... Acta neuropathologica 126, 385-399, 2013 | 422 | 2013 |
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Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping LV Gushchina, EC Frair, N Rohan, AJ Bradley, TR Simmons, HD Chavan, ... Human Gene Therapy 32 (17-18), 882-894, 2021 | 38 | 2021 |
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A synthetic biology approach to probing nucleosome symmetry Y Ichikawa, CF Connelly, A Appleboim, TCR Miller, H Jacobi, NA Abshiru, ... elife 6, e28836, 2017 | 27 | 2017 |
Normalization benchmark of ATAC-seq datasets shows the importance of accounting for GC-content effects K Van den Berge, HJ Chou, HR de Bézieux, K Street, D Risso, J Ngai, ... Cell Reports Methods 2 (11), 2022 | 11 | 2022 |
AT845 gene replacement therapy for late onset Pompe disease: An update on safety and preliminary efficacy data from FORTIS, a phase I/II open-label clinical study JD Manera, T Mozaffar, JW Day, N Longo, V Straub, F Varfaj, HJ Chou, ... Molecular Genetics and Metabolism 138 (2), 107078, 2023 | 4 | 2023 |
High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy G Andreoletti, O Romano, HJ Chou, MJ Sefid-Dashti, A Grilli, C Chen, ... The American Journal of Human Genetics 110 (10), 1648-1660, 2023 | 1 | 2023 |
A Latent Activated Olfactory Stem Cell State Revealed by Single Cell Transcriptomic and Epigenomic Profiling K Van den Berge, HJ Chou, D Kunda, D Risso, K Street, E Purdom, ... bioRxiv, 2023 | | 2023 |