715 related articles for article (PubMed ID: 24870228)
1. 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]
2. 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]
3. Gene therapy: Repair and replace.
Fischer A
Nature; 2014 Jun; 510(7504):226-7. PubMed ID: 24870243
[No Abstract] [Full Text] [Related]
4. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.
Schiroli G; Ferrari S; Conway A; Jacob A; Capo V; Albano L; Plati T; Castiello MC; Sanvito F; Gennery AR; Bovolenta C; Palchaudhuri R; Scadden DT; Holmes MC; Villa A; Sitia G; Lombardo A; Genovese P; Naldini L
Sci Transl Med; 2017 Oct; 9(411):. PubMed ID: 29021165
[TBL] [Abstract][Full Text] [Related]
5. Nuclease-free Adeno-Associated Virus-Mediated Il2rg Gene Editing in X-SCID Mice.
Hiramoto T; Li LB; Funk SE; Hirata RK; Russell DW
Mol Ther; 2018 May; 26(5):1255-1265. PubMed ID: 29606506
[TBL] [Abstract][Full Text] [Related]
6. CD105 is a surface marker for receptor-targeted gene transfer into human long-term repopulating hematopoietic stem cells.
Kays SK; Kaufmann KB; Abel T; Brendel C; Bonig H; Grez M; Buchholz CJ; Kneissl S
Stem Cells Dev; 2015 Mar; 24(6):714-23. PubMed ID: 25517513
[TBL] [Abstract][Full Text] [Related]
7. Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.
Alzubi J; Pallant C; Mussolino C; Howe SJ; Thrasher AJ; Cathomen T
Sci Rep; 2017 Sep; 7(1):12475. PubMed ID: 28963568
[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. Comparison of Different Cytokine Conditions Reveals Resveratrol as a New Molecule for Ex Vivo Cultivation of Cord Blood-Derived Hematopoietic Stem Cells.
Heinz N; Ehrnström B; Schambach A; Schwarzer A; Modlich U; Schiedlmeier B
Stem Cells Transl Med; 2015 Sep; 4(9):1064-72. PubMed ID: 26160960
[TBL] [Abstract][Full Text] [Related]
10. Mesenchymal stem cells secreting angiopoietin-like-5 support efficient expansion of human hematopoietic stem cells without compromising their repopulating potential.
Khoury M; Drake A; Chen Q; Dong D; Leskov I; Fragoso MF; Li Y; Iliopoulou BP; Hwang W; Lodish HF; Chen J
Stem Cells Dev; 2011 Aug; 20(8):1371-81. PubMed ID: 21142526
[TBL] [Abstract][Full Text] [Related]
11. Pretransplant mobilization with granulocyte colony-stimulating factor improves B-cell reconstitution by lentiviral vector gene therapy in SCID-X1 mice.
Huston MW; Riegman AR; Yadak R; van Helsdingen Y; de Boer H; van Til NP; Wagemaker G
Hum Gene Ther; 2014 Oct; 25(10):905-14. PubMed ID: 25222508
[TBL] [Abstract][Full Text] [Related]
12. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz E; Zhou S; Lockey T; Abdelsamed H; Cross SJ; Kang G; Ma Z; Condori J; Dowdy J; Triplett B; Li C; Maron G; Aldave Becerra JC; Church JA; Dokmeci E; Love JT; da Matta Ain AC; van der Watt H; Tang X; Janssen W; Ryu BY; De Ravin SS; Weiss MJ; Youngblood B; Long-Boyle JR; Gottschalk S; Meagher MM; Malech HL; Puck JM; Cowan MJ; Sorrentino BP
N Engl J Med; 2019 Apr; 380(16):1525-1534. PubMed ID: 30995372
[TBL] [Abstract][Full Text] [Related]
13. Human CD34+ hematopoietic stem cells capable of multilineage engrafting NOD/SCID mice express flt3: distinct flt3 and c-kit expression and response patterns on mouse and candidate human hematopoietic stem cells.
Sitnicka E; Buza-Vidas N; Larsson S; Nygren JM; Liuba K; Jacobsen SE
Blood; 2003 Aug; 102(3):881-6. PubMed ID: 12676789
[TBL] [Abstract][Full Text] [Related]
14. CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
Brault J; Liu T; Liu S; Lawson A; Choi U; Kozhushko N; Bzhilyanskaya V; Pavel-Dinu M; Meis RJ; Eckhaus MA; Burkett SS; Bosticardo M; Kleinstiver BP; Notarangelo LD; Lazzarotto CR; Tsai SQ; Wu X; Dahl GA; Porteus MH; Malech HL; De Ravin SS
Front Immunol; 2022; 13():1067417. PubMed ID: 36685559
[TBL] [Abstract][Full Text] [Related]
15. Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1.
McIvor RS; Eaton EJ; Webber BR; Moriarity BS
Mol Ther; 2024 Jun; 32(6):1606-1608. PubMed ID: 38781958
[No Abstract] [Full Text] [Related]
16. Genome editing: a tool for research and therapy: targeted genome editing hits the clinic.
Lombardo A; Naldini L
Nat Med; 2014 Oct; 20(10):1101-3. PubMed ID: 25295939
[No Abstract] [Full Text] [Related]
17. Therapeutic base editing of human hematopoietic stem cells.
Zeng J; Wu Y; Ren C; Bonanno J; Shen AH; Shea D; Gehrke JM; Clement K; Luk K; Yao Q; Kim R; Wolfe SA; Manis JP; Pinello L; Joung JK; Bauer DE
Nat Med; 2020 Apr; 26(4):535-541. PubMed ID: 32284612
[TBL] [Abstract][Full Text] [Related]
18. Therapeutic gene editing in CD34
Diez B; Genovese P; Roman-Rodriguez FJ; Alvarez L; Schiroli G; Ugalde L; Rodriguez-Perales S; Sevilla J; Diaz de Heredia C; Holmes MC; Lombardo A; Naldini L; Bueren JA; Rio P
EMBO Mol Med; 2017 Nov; 9(11):1574-1588. PubMed ID: 28899930
[TBL] [Abstract][Full Text] [Related]
19. Safety and efficacy of ex vivo expanded CD34
Zhang Y; Shen B; Guan X; Qin M; Ren Z; Ma Y; Dai W; Ding X; Jiang Y
Stem Cell Res Ther; 2019 Jun; 10(1):173. PubMed ID: 31196160
[TBL] [Abstract][Full Text] [Related]
20. Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system.
Sumiyoshi T; Holt NG; Hollis RP; Ge S; Cannon PM; Crooks GM; Kohn DB
Hum Gene Ther; 2009 Dec; 20(12):1607-26. PubMed ID: 19689196
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]