199 related articles for article (PubMed ID: 36685559)
1. 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]
2. Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration.
Bloomer H; Smith RH; Hakami W; Larochelle A
Mol Ther; 2021 Apr; 29(4):1611-1624. PubMed ID: 33309880
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.
Brault J; Liu T; Bello E; Liu S; Sweeney CL; Meis RJ; Koontz S; Corsino C; Choi U; Vayssiere G; Bosticardo M; Dowdell K; Lazzarotto CR; Clark AB; Notarangelo LD; Ravell JC; Lenardo MJ; Kleinstiver BP; Tsai SQ; Wu X; Dahl GA; Malech HL; De Ravin SS
Blood; 2021 Dec; 138(26):2768-2780. PubMed ID: 34086870
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing.
Hou Y; Ureña-Bailén G; Mohammadian Gol T; Gratz PG; Gratz HP; Roig-Merino A; Antony JS; Lamsfus-Calle A; Daniel-Moreno A; Handgretinger R; Mezger M
Genes (Basel); 2022 Dec; 13(12):. PubMed ID: 36553615
[TBL] [Abstract][Full Text] [Related]
7. 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]
8. Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model.
Avilés Mendoza GJ; Seidel NE; Otsu M; Anderson SM; Simon-Stoos K; Herrera A; Hoogstraten-Miller S; Malech HL; Candotti F; Puck JM; Bodine DM
Mol Ther; 2001 Apr; 3(4):565-73. PubMed ID: 11319919
[TBL] [Abstract][Full Text] [Related]
9. Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs.
Iancu O; Allen D; Knop O; Zehavi Y; Breier D; Arbiv A; Lev A; Lee YN; Beider K; Nagler A; Somech R; Hendel A
Mol Ther Nucleic Acids; 2023 Mar; 31():105-121. PubMed ID: 36618262
[TBL] [Abstract][Full Text] [Related]
10. Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for
Dudek AM; Porteus MH
Front Immunol; 2021; 12():660302. PubMed ID: 34122418
[TBL] [Abstract][Full Text] [Related]
11. CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells.
Rai R; Steinberg Z; Romito M; Zinghirino F; Hu YT; White N; Naseem A; Thrasher AJ; Turchiano G; Cavazza A
Hum Gene Ther; 2024 Apr; 35(7-8):269-283. PubMed ID: 38251667
[TBL] [Abstract][Full Text] [Related]
12. CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease.
De Ravin SS; Li L; Wu X; Choi U; Allen C; Koontz S; Lee J; Theobald-Whiting N; Chu J; Garofalo M; Sweeney C; Kardava L; Moir S; Viley A; Natarajan P; Su L; Kuhns D; Zarember KA; Peshwa MV; Malech HL
Sci Transl Med; 2017 Jan; 9(372):. PubMed ID: 28077679
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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]
15. Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Poletti V; Charrier S; Corre G; Gjata B; Vignaud A; Zhang F; Rothe M; Schambach A; Gaspar HB; Thrasher AJ; Mavilio F
Mol Ther Methods Clin Dev; 2018 Jun; 9():257-269. PubMed ID: 29707600
[TBL] [Abstract][Full Text] [Related]
16. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
Lattanzi A; Meneghini V; Pavani G; Amor F; Ramadier S; Felix T; Antoniani C; Masson C; Alibeu O; Lee C; Porteus MH; Bao G; Amendola M; Mavilio F; Miccio A
Mol Ther; 2019 Jan; 27(1):137-150. PubMed ID: 30424953
[TBL] [Abstract][Full Text] [Related]
17. CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia.
Tran NT; Graf R; Wulf-Goldenberg A; Stecklum M; Strauß G; Kühn R; Kocks C; Rajewsky K; Chu VT
Mol Ther; 2020 Dec; 28(12):2621-2634. PubMed ID: 32822592
[TBL] [Abstract][Full Text] [Related]
18. Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex.
de Alencastro G; Puzzo F; Pavel-Dinu M; Zhang F; Pillay S; Majzoub K; Tiffany M; Jang H; Sheikali A; Cromer MK; Meetei R; Carette JE; Porteus MH; Pekrun K; Kay MA
Mol Ther; 2021 Mar; 29(3):1016-1027. PubMed ID: 33678249
[TBL] [Abstract][Full Text] [Related]
19. Efficient CRISPR/Cas9-Mediated Gene Knockin in Mouse Hematopoietic Stem and Progenitor Cells.
Tran NT; Sommermann T; Graf R; Trombke J; Pempe J; Petsch K; Kühn R; Rajewsky K; Chu VT
Cell Rep; 2019 Sep; 28(13):3510-3522.e5. PubMed ID: 31553918
[TBL] [Abstract][Full Text] [Related]
20. Genome Engineering of Hematopoietic Stem Cells Using CRISPR/Cas9 System.
Devaraju N; Rajendiran V; Ravi NS; Mohankumar KM
Methods Mol Biol; 2022; 2429():307-331. PubMed ID: 35507170
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
