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148 results

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Page 1
Long somatic DNA-repeat expansion drives neurodegeneration in Huntington's disease.
Handsaker RE, Kashin S, Reed NM, Tan S, Lee WS, McDonald TM, Morris K, Kamitaki N, Mullally CD, Morakabati NR, Goldman M, Lind G, Kohli R, Lawton E, Hogan M, Ichihara K, Berretta S, McCarroll SA. Handsaker RE, et al. Among authors: lee ws. Cell. 2025 Feb 6;188(3):623-639.e19. doi: 10.1016/j.cell.2024.11.038. Epub 2025 Jan 16. Cell. 2025. PMID: 39824182 Free PMC article.
Antisense oligonucleotide-mediated MSH3 suppression reduces somatic CAG repeat expansion in Huntington's disease iPSC-derived striatal neurons.
Bunting EL, Donaldson J, Cumming SA, Olive J, Broom E, Miclăuș M, Hamilton J, Tegtmeyer M, Zhao HT, Brenton J, Lee WS, Handsaker RE, Li S, Ford B, Ryten M, McCarroll SA, Kordasiewicz HB, Monckton DG, Balmus G, Flower M, Tabrizi SJ. Bunting EL, et al. Among authors: lee ws. Sci Transl Med. 2025 Feb 12;17(785):eadn4600. doi: 10.1126/scitranslmed.adn4600. Epub 2025 Feb 12. Sci Transl Med. 2025. PMID: 39937881
Literature-based predictions of Mendelian disease therapies.
Deisseroth CA, Lee WS, Kim J, Jeong HH, Dhindsa RS, Wang J, Zoghbi HY, Liu Z. Deisseroth CA, et al. Among authors: lee ws. Am J Hum Genet. 2023 Oct 5;110(10):1661-1672. doi: 10.1016/j.ajhg.2023.08.018. Epub 2023 Sep 22. Am J Hum Genet. 2023. PMID: 37741276 Free PMC article.
A shared DNA-repeat toxicity threshold, reached somatically at cell-type-specific rates, unites cortical and striatal neurodegeneration in Huntington's disease.
Kashin S, Lee WS, McDonald TM, Morris K, Handsaker RE, Mello C, Spina L, Reed NM, de Rivera H, Jana SH, Hogan M, Berretta S, McCarroll SA. Kashin S, et al. Among authors: lee ws. bioRxiv [Preprint]. 2025 Dec 11:2025.12.09.688862. doi: 10.64898/2025.12.09.688862. bioRxiv. 2025. PMID: 41427302 Free PMC article. Preprint.
148 results