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Wang H, Georgakopoulou A, Psatha N, Li C, Capsali C, Samal HB, Anagnostopoulos A, Ehrhardt A, Izsvak Z, Papayannopoulou T, Lieber A. (2019) In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia. J Clin Invest.129(2):598-615.
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Li C, Course MM, McNeish IA, Drescher CW, Valdmanis PN, Lieber A. (2020) Prophylactic in vivo hematopoietic stem cell gene therapy with an immune checkpoint inhibitor reverses tumor growth in syngeneic mouse tumor models. Cancer Res. 80(3):549-560.
Wang H, Georgakopoulou A, Li C, Liu Z, Gil S, Bashyam A, Yannaki E, Anagnostopoulos A, Pande A, Izsvák Z, Papayannopoulou T, Lieber A. (2020) Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements. JCI Insights. 5(16):e139538.
Li C, Wang H, Georgakopoulou A, Gil S, Yannaki E, Lieber A. (2020) In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model. Mol Ther. 2020 Sep 5:S1525-0016(20)30458-5.
Li C, Georgakopoulou A, Mishra A, Gil S, Hawkins RD, Yannaki E, Lieber A. (2021) In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal g-globin in β-YAC mice. Blood Adv. 5(4):1122-1135.
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