361 related articles for article (PubMed ID: 36553615)
1. 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]
2. 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]
3. Generation of novel Il2rg-knockout mice with clustered regularly interspaced short palindromic repeats (CRISPR) and Cas9.
Byambaa S; Uosaki H; Hara H; Nagao Y; Abe T; Shibata H; Nureki O; Ohmori T; Hanazono Y
Exp Anim; 2020 Apr; 69(2):189-198. PubMed ID: 31801915
[TBL] [Abstract][Full Text] [Related]
4. Mosaicism in CRISPR/Cas9-mediated genome editing.
Mehravar M; Shirazi A; Nazari M; Banan M
Dev Biol; 2019 Jan; 445(2):156-162. PubMed ID: 30359560
[TBL] [Abstract][Full Text] [Related]
5. 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]
6. 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]
7. Late-Onset Combined Immunodeficiency with a Novel IL2RG Mutation and Probable Revertant Somatic Mosaicism.
Okuno Y; Hoshino A; Muramatsu H; Kawashima N; Wang X; Yoshida K; Wada T; Gunji M; Toma T; Kato T; Shiraishi Y; Iwata A; Hori T; Kitoh T; Chiba K; Tanaka H; Sanada M; Takahashi Y; Nonoyama S; Ito M; Miyano S; Ogawa S; Kojima S; Kanegane H
J Clin Immunol; 2015 Oct; 35(7):610-4. PubMed ID: 26407811
[TBL] [Abstract][Full Text] [Related]
8. CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders.
Ugalde L; Fañanas S; Torres R; Quintana-Bustamante O; Río P
Cytotherapy; 2023 Mar; 25(3):277-285. PubMed ID: 36610813
[TBL] [Abstract][Full Text] [Related]
9. Gene Therapy with CRISPR/Cas9 Coming to Age for HIV Cure.
Soriano V
AIDS Rev; 2017; 19(3):167-172. PubMed ID: 29019352
[TBL] [Abstract][Full Text] [Related]
10. CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation.
Ma L; Jang L; Chen J; Song J; Yang D; Zhang J; Chen YE; Xu J
J Vis Exp; 2019 Jun; (148):. PubMed ID: 31282887
[TBL] [Abstract][Full Text] [Related]
11. Efficient gene correction of an aberrant splice site in β-thalassaemia iPSCs by CRISPR/Cas9 and single-strand oligodeoxynucleotides.
Xiong Z; Xie Y; Yang Y; Xue Y; Wang D; Lin S; Chen D; Lu D; He L; Song B; Yang Y; Sun X
J Cell Mol Med; 2019 Dec; 23(12):8046-8057. PubMed ID: 31631510
[TBL] [Abstract][Full Text] [Related]
12. Prime Editing for Human Gene Therapy: Where Are We Now?
Godbout K; Tremblay JP
Cells; 2023 Feb; 12(4):. PubMed ID: 36831203
[TBL] [Abstract][Full Text] [Related]
13. Somatic Reversion of a Novel
Hou Y; Gratz HP; Ureña-Bailén G; Gratz PG; Schilbach-Stückle K; Renno T; Güngör D; Mader DA; Malenke E; Antony JS; Handgretinger R; Mezger M
Genes (Basel); 2021 Dec; 13(1):. PubMed ID: 35052377
[TBL] [Abstract][Full Text] [Related]
14. 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]
15. Case Report: A Novel IL2RG Frame-Restoring Rescue Mutation Mimics Early T Cell Engraftment Following Haploidentical Hematopoietic Stem Cell Transplantation in a Patient With X-SCID.
Steininger J; Leiss-Piller A; Geier CB; Rossmanith R; Elfeky R; Bra D; Pichler H; Lawitschka A; Zubarovskaya N; Artacker G; Matthes-Leodolter S; Eibl MM; Wolf HM
Front Immunol; 2021; 12():644687. PubMed ID: 33959125
[TBL] [Abstract][Full Text] [Related]
16. Suppression of mosaic mutation by co-delivery of CRISPR associated protein 9 and three-prime repair exonuclease 2 into porcine zygotes via electroporation.
Yamashita S; Kogasaka Y; Hiradate Y; Tanemura K; Sendai Y
J Reprod Dev; 2020 Feb; 66(1):41-48. PubMed ID: 31761839
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. 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]
19. Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases.
Hansen S; McClements ME; Corydon TJ; MacLaren RE
Cells; 2023 Jan; 12(3):. PubMed ID: 36766782
[TBL] [Abstract][Full Text] [Related]
20. CRISPR/Cas gene editing in the human germline.
Bekaert B; Boel A; Cosemans G; De Witte L; Menten B; Heindryckx B
Semin Cell Dev Biol; 2022 Nov; 131():93-107. PubMed ID: 35305903
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]