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In vivo HSC prime editing rescues sickle cell disease in a mouse model.
Li C, Georgakopoulou A, Newby GA, Chen PJ, Everette KA, Paschoudi K, Vlachaki E, Gil S, Anderson AK, Koob T, Huang L, Wang H, Kiem HP, Liu DR, Yannaki E, Lieber A. Li C, et al. Blood. 2023 Apr 27;141(17):2085-2099. doi: 10.1182/blood.2022018252. Blood. 2023. PMID: 36800642 Free PMC article.
A Combined In Vivo HSC Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice.
Wang H, Richter M, Psatha N, Li C, Kim J, Liu J, Ehrhardt A, Nilsson SK, Cao B, Palmer D, Ng P, Izsvák Z, Haworth KG, Kiem HP, Papayannopoulou T, Lieber A. Wang H, et al. Among authors: li c. Mol Ther Methods Clin Dev. 2017 Nov 10;8:52-64. doi: 10.1016/j.omtm.2017.11.004. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29255741 Free PMC article.
In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia.
Wang H, Georgakopoulou A, Psatha N, Li C, Capsali C, Samal HB, Anagnostopoulos A, Ehrhardt A, Izsvák Z, Papayannopoulou T, Yannaki E, Lieber A. Wang H, et al. Among authors: li c. J Clin Invest. 2019 Feb 1;129(2):598-615. doi: 10.1172/JCI122836. Epub 2018 Dec 18. J Clin Invest. 2019. PMID: 30422819 Free PMC article.
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