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6. Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles. Yu KR; Natanson H; Dunbar CE Hum Gene Ther; 2016 Oct; 27(10):729-740. PubMed ID: 27483988 [TBL] [Abstract][Full Text] [Related]
7. CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report. Wang X; Raghavan A; Chen T; Qiao L; Zhang Y; Ding Q; Musunuru K Arterioscler Thromb Vasc Biol; 2016 May; 36(5):783-6. PubMed ID: 26941020 [TBL] [Abstract][Full Text] [Related]
8. Restoring T and B cell generation in X-linked severe combined immunodeficiency mice through hematopoietic stem cells adenine base editing. Zhang L; Li K; Liu Z; An L; Wei H; Pang S; Cao Z; Huang X; Jin X; Ma X Mol Ther; 2024 Jun; 32(6):1658-1671. PubMed ID: 38532630 [TBL] [Abstract][Full Text] [Related]
9. Targeted genome editing in human repopulating haematopoietic stem cells. Genovese P; Schiroli G; Escobar G; Tomaso TD; Firrito C; Calabria A; Moi D; Mazzieri R; Bonini C; Holmes MC; Gregory PD; van der Burg M; Gentner B; Montini E; Lombardo A; Naldini L Nature; 2014 Jun; 510(7504):235-240. PubMed ID: 24870228 [TBL] [Abstract][Full Text] [Related]
10. Genome editing for human gene therapy. Meissner TB; Mandal PK; Ferreira LM; Rossi DJ; Cowan CA Methods Enzymol; 2014; 546():273-95. PubMed ID: 25398345 [TBL] [Abstract][Full Text] [Related]
11. Gene correction for SCID-X1 in long-term hematopoietic stem cells. Pavel-Dinu M; Wiebking V; Dejene BT; Srifa W; Mantri S; Nicolas CE; Lee C; Bao G; Kildebeck EJ; Punjya N; Sindhu C; Inlay MA; Saxena N; DeRavin SS; Malech H; Roncarolo MG; Weinberg KI; Porteus MH Nat Commun; 2019 Apr; 10(1):1634. PubMed ID: 30967552 [TBL] [Abstract][Full Text] [Related]
12. The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice. Ou Z; Niu X; He W; Chen Y; Song B; Xian Y; Fan D; Tang D; Sun X Sci Rep; 2016 Sep; 6():32463. PubMed ID: 27581487 [TBL] [Abstract][Full Text] [Related]
13. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response. Schiroli G; Conti A; Ferrari S; Della Volpe L; Jacob A; Albano L; Beretta S; Calabria A; Vavassori V; Gasparini P; Salataj E; Ndiaye-Lobry D; Brombin C; Chaumeil J; Montini E; Merelli I; Genovese P; Naldini L; Di Micco R Cell Stem Cell; 2019 Apr; 24(4):551-565.e8. PubMed ID: 30905619 [TBL] [Abstract][Full Text] [Related]
18. Combined lentiviral- and RNA-mediated CRISPR/Cas9 delivery for efficient and traceable gene editing in human hematopoietic stem and progenitor cells. Yudovich D; Bäckström A; Schmiderer L; Žemaitis K; Subramaniam A; Larsson J Sci Rep; 2020 Dec; 10(1):22393. PubMed ID: 33372184 [TBL] [Abstract][Full Text] [Related]
19. Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing. Wen J; Tao W; Hao S; Zu Y J Hematol Oncol; 2017 Jun; 10(1):119. PubMed ID: 28610635 [TBL] [Abstract][Full Text] [Related]
20. Gene targeting technologies in rats: zinc finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats. Mashimo T Dev Growth Differ; 2014 Jan; 56(1):46-52. PubMed ID: 24372523 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]