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Page 1
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: georgakopoulou a. 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.
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.
Li C, Wang H, Gil S, Germond A, Fountain C, Baldessari A, Kim J, Liu Z, Georgakopoulou A, Radtke S, Raskó T, Pande A, Chiang C, Chin E, Yannaki E, Izsvák Z, Papayannopoulou T, Kiem HP, Lieber A. Li C, et al. Among authors: georgakopoulou a. Mol Ther Methods Clin Dev. 2021 Dec 6;24:127-141. doi: 10.1016/j.omtm.2021.12.003. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 35036470 Free PMC article.
Erratum: Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors.
Li C, Wang H, Gil S, Germond A, Fountain C, Baldessari A, Kim J, Liu Z, Georgakopoulou A, Radtke S, Raskó T, Pande A, Chiang C, Chin E, Yannaki E, Izsvák Z, Papayannopoulou T, Kiem HP, Lieber A. Li C, et al. Among authors: georgakopoulou a. Mol Ther Methods Clin Dev. 2022 May 22;25:533. doi: 10.1016/j.omtm.2022.05.003. eCollection 2022 Jun 9. Mol Ther Methods Clin Dev. 2022. PMID: 35662812 Free PMC article.
In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.
Li C, Georgakopoulou A, Newby GA, Everette KA, Nizamis E, 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. Among authors: georgakopoulou a. JCI Insight. 2022 Oct 10;7(19):e162939. doi: 10.1172/jci.insight.162939. JCI Insight. 2022. PMID: 36006707 Free PMC article.
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. Among authors: georgakopoulou a. Blood. 2023 Apr 27;141(17):2085-2099. doi: 10.1182/blood.2022018252. Blood. 2023. PMID: 36800642 Free PMC article.
26 results