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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.
Chang KH, Smith SE, Sullivan T, Chen K, Zhou Q, West JA, Liu M, Liu Y, Vieira BF, Sun C, Hong VP, Zhang M, Yang X, Reik A, Urnov FD, Rebar EJ, Holmes MC, Danos O, Jiang H, Tan S. Chang KH, et al. Among authors: sun c. Mol Ther Methods Clin Dev. 2017 Jan 11;4:137-148. doi: 10.1016/j.omtm.2016.12.009. eCollection 2017 Mar 17. Mol Ther Methods Clin Dev. 2017. PMID: 28344999 Free PMC article.
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model.
Chen YA, Kankel MW, Hana S, Lau SK, Zavodszky MI, McKissick O, Mastrangelo N, Dion J, Wang B, Ferretti D, Koske D, Lehman S, Koszka K, McLaughlin H, Liu M, Marshall E, Fabian AJ, Cullen P, Marsh G, Hamann S, Craft M, Sebalusky J, Arnold HM, Driscoll R, Sheehy A, Luo Y, Manca S, Carlile T, Sun C, Sigrist K, McCampbell A, Henderson CE, Lo SC. Chen YA, et al. Among authors: sun c. Gene Ther. 2023 May;30(5):443-454. doi: 10.1038/s41434-022-00375-w. Epub 2022 Dec 1. Gene Ther. 2023. PMID: 36450833 Free PMC article.
Parkinson's disease associated with pure ATXN10 repeat expansion.
Schüle B, McFarland KN, Lee K, Tsai YC, Nguyen KD, Sun C, Liu M, Byrne C, Gopi R, Huang N, Langston JW, Clark T, Gil FJJ, Ashizawa T. Schüle B, et al. Among authors: sun c. NPJ Parkinsons Dis. 2017 Sep 5;3:27. doi: 10.1038/s41531-017-0029-x. eCollection 2017. NPJ Parkinsons Dis. 2017. PMID: 28890930 Free PMC article.
Organotypic Brain Slice Culture Microglia Exhibit Molecular Similarity to Acutely-Isolated Adult Microglia and Provide a Platform to Study Neuroinflammation.
Delbridge ARD, Huh D, Brickelmaier M, Burns JC, Roberts C, Challa R, Raymond N, Cullen P, Carlile TM, Ennis KA, Liu M, Sun C, Allaire NE, Foos M, Tsai HH, Franchimont N, Ransohoff RM, Butts C, Mingueneau M. Delbridge ARD, et al. Among authors: sun c. Front Cell Neurosci. 2020 Dec 21;14:592005. doi: 10.3389/fncel.2020.592005. eCollection 2020. Front Cell Neurosci. 2020. PMID: 33473245 Free PMC article.
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice.
Hana S, Peterson M, McLaughlin H, Marshall E, Fabian AJ, McKissick O, Koszka K, Marsh G, Craft M, Xu S, Sorets A, Torregrosa T, Sun C, Henderson CE, Lo SC. Hana S, et al. Among authors: sun c. Gene Ther. 2021 Nov;28(10-11):646-658. doi: 10.1038/s41434-021-00224-2. Epub 2021 Feb 8. Gene Ther. 2021. PMID: 33558692 Free PMC article.
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