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Journal Abstract Search

356 related items for PubMed ID: 31773990

  • 1. Differential Transgene Silencing of Myeloid-Specific Promoters in the AAVS1 Safe Harbor Locus of Induced Pluripotent Stem Cell-Derived Myeloid Cells.
    Klatt D, Cheng E, Hoffmann D, Santilli G, Thrasher AJ, Brendel C, Schambach A.
    Hum Gene Ther; 2020 Feb; 31(3-4):199-210. PubMed ID: 31773990
    [Abstract] [Full Text] [Related]

  • 2. TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.
    Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, Schambach A, Cathomen T.
    Biomaterials; 2015 Nov; 69():191-200. PubMed ID: 26295532
    [Abstract] [Full Text] [Related]

  • 3. Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers.
    Hong SG, Yada RC, Choi K, Carpentier A, Liang TJ, Merling RK, Sweeney CL, Malech HL, Jung M, Corat MAF, AlJanahi AA, Lin Y, Liu H, Tunc I, Wang X, Palisoc M, Pittaluga S, Boehm M, Winkler T, Zou J, Dunbar CE.
    Mol Ther; 2017 Jan 04; 25(1):44-53. PubMed ID: 28129126
    [Abstract] [Full Text] [Related]

  • 4. Variable expression and silencing of CRISPR-Cas9 targeted transgenes identifies the AAVS1 locus as not an entirely safe harbour.
    Bhagwan JR, Collins E, Mosqueira D, Bakar M, Johnson BB, Thompson A, Smith JGW, Denning C.
    F1000Res; 2019 Jan 04; 8():1911. PubMed ID: 32789000
    [Abstract] [Full Text] [Related]

  • 5. Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells.
    Santilli G, Almarza E, Brendel C, Choi U, Beilin C, Blundell MP, Haria S, Parsley KL, Kinnon C, Malech HL, Bueren JA, Grez M, Thrasher AJ.
    Mol Ther; 2011 Jan 04; 19(1):122-32. PubMed ID: 20978475
    [Abstract] [Full Text] [Related]

  • 6. Stable enhanced green fluorescent protein expression after differentiation and transplantation of reporter human induced pluripotent stem cells generated by AAVS1 transcription activator-like effector nucleases.
    Luo Y, Liu C, Cerbini T, San H, Lin Y, Chen G, Rao MS, Zou J.
    Stem Cells Transl Med; 2014 Jul 04; 3(7):821-35. PubMed ID: 24833591
    [Abstract] [Full Text] [Related]

  • 7. Targeted transgene insertion into the AAVS1 locus driven by baculoviral vector-mediated zinc finger nuclease expression in human-induced pluripotent stem cells.
    Tay FC, Tan WK, Goh SL, Ramachandra CJ, Lau CH, Zhu H, Chen C, Du S, Phang RZ, Shahbazi M, Fan W, Wang S.
    J Gene Med; 2013 Oct 04; 15(10):384-95. PubMed ID: 24105820
    [Abstract] [Full Text] [Related]

  • 8. Targeted Repair of CYBB in X-CGD iPSCs Requires Retention of Intronic Sequences for Expression and Functional Correction.
    Sweeney CL, Zou J, Choi U, Merling RK, Liu A, Bodansky A, Burkett S, Kim JW, De Ravin SS, Malech HL.
    Mol Ther; 2017 Feb 01; 25(2):321-330. PubMed ID: 28153086
    [Abstract] [Full Text] [Related]

  • 9. Engineering the AAVS1 locus for consistent and scalable transgene expression in human iPSCs and their differentiated derivatives.
    Oceguera-Yanez F, Kim SI, Matsumoto T, Tan GW, Xiang L, Hatani T, Kondo T, Ikeya M, Yoshida Y, Inoue H, Woltjen K.
    Methods; 2016 May 15; 101():43-55. PubMed ID: 26707206
    [Abstract] [Full Text] [Related]

  • 10. Targeting the AAVS1 Site by CRISPR/Cas9 with an Inducible Transgene Cassette for the Neuronal Differentiation of Human Pluripotent Stem Cells.
    Gu J, Rollo B, Sumer H, Cromer B.
    Methods Mol Biol; 2022 May 15; 2495():99-114. PubMed ID: 35696030
    [Abstract] [Full Text] [Related]

  • 11. Human miR223 promoter as a novel myelo-specific promoter for chronic granulomatous disease gene therapy.
    Brendel C, Hänseler W, Wohlgensinger V, Bianchi M, Tokmak S, Chen-Wichmann L, Kuzmenko E, Cesarovic N, Nicholls F, Reichenbach J, Seger R, Grez M, Siler U.
    Hum Gene Ther Methods; 2013 Jun 15; 24(3):151-9. PubMed ID: 23489116
    [Abstract] [Full Text] [Related]

  • 12. An AAVS1-targeted minigene platform for correction of iPSCs from all five types of chronic granulomatous disease.
    Merling RK, Sweeney CL, Chu J, Bodansky A, Choi U, Priel DL, Kuhns DB, Wang H, Vasilevsky S, De Ravin SS, Winkler T, Dunbar CE, Zou J, Zarember KA, Gallin JI, Holland SM, Malech HL.
    Mol Ther; 2015 Jan 15; 23(1):147-57. PubMed ID: 25288370
    [Abstract] [Full Text] [Related]

  • 13. Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.
    Klatt D, Cheng E, Philipp F, Selich A, Dahlke J, Schmidt RE, Schott JW, Büning H, Hoffmann D, Thrasher AJ, Schambach A.
    Stem Cell Reports; 2019 Oct 08; 13(4):590-598. PubMed ID: 31543470
    [Abstract] [Full Text] [Related]

  • 14. A ubiquitous chromatin opening element prevents transgene silencing in pluripotent stem cells and their differentiated progeny.
    Pfaff N, Lachmann N, Ackermann M, Kohlscheen S, Brendel C, Maetzig T, Niemann H, Antoniou MN, Grez M, Schambach A, Cantz T, Moritz T.
    Stem Cells; 2013 Mar 08; 31(3):488-99. PubMed ID: 23307570
    [Abstract] [Full Text] [Related]

  • 15. A Doxycycline-Inducible System for Genetic Correction of iPSC Disease Models.
    Sim X, Cardenas-Diaz FL, French DL, Gadue P.
    Methods Mol Biol; 2016 Mar 08; 1353():13-23. PubMed ID: 25630922
    [Abstract] [Full Text] [Related]

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  • 19. Transfection, selection, and colony-picking of human induced pluripotent stem cells TALEN-targeted with a GFP gene into the AAVS1 safe harbor.
    Cerbini T, Luo Y, Rao MS, Zou J.
    J Vis Exp; 2015 Feb 01; (96):. PubMed ID: 25741760
    [Abstract] [Full Text] [Related]

  • 20. Generation and characterization of a human iPSC cell line expressing inducible Cas9 in the "safe harbor" AAVS1 locus.
    Castaño J, Bueno C, Jiménez-Delgado S, Roca-Ho H, Fraga MF, Fernandez AF, Nakanishi M, Torres-Ruiz R, Rodríguez-Perales S, Menéndez P.
    Stem Cell Res; 2017 May 01; 21():137-140. PubMed ID: 28677529
    [Abstract] [Full Text] [Related]

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