148 related articles for article (PubMed ID: 38134938)
21. Generation of an in vitro model of β-thalassemia using the CRISPR/Cas9 genome editing system.
Ajami M; Atashi A; Kaviani S; Kiani J; Soleimani M
J Cell Biochem; 2020 Feb; 121(2):1420-1430. PubMed ID: 31596028
[TBL] [Abstract][Full Text] [Related]
22. Genetic correction of concurrent α- and β-thalassemia patient-derived pluripotent stem cells by the CRISPR-Cas9 technology.
Li L; Yi H; Liu Z; Long P; Pan T; Huang Y; Li Y; Li Q; Ma Y
Stem Cell Res Ther; 2022 Mar; 13(1):102. PubMed ID: 35255977
[TBL] [Abstract][Full Text] [Related]
23. Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.
Persons DA; Allay ER; Sawai N; Hargrove PW; Brent TP; Hanawa H; Nienhuis AW; Sorrentino BP
Blood; 2003 Jul; 102(2):506-13. PubMed ID: 12663444
[TBL] [Abstract][Full Text] [Related]
24. The safety and effectiveness of genetically corrected iPSCs derived from β-thalassaemia patients in nonmyeloablative β-thalassaemic mice.
Xian Y; Xie Y; Song B; Ou Z; Ouyang S; Xie Y; Yang Y; Xiong Z; Li H; Sun X
Stem Cell Res Ther; 2020 Jul; 11(1):288. PubMed ID: 32678022
[TBL] [Abstract][Full Text] [Related]
25. Genome Editing for the β-Hemoglobinopathies.
Porteus MH
Adv Exp Med Biol; 2017; 1013():203-217. PubMed ID: 29127682
[TBL] [Abstract][Full Text] [Related]
26. Reversal of aberrant splicing of beta-thalassaemia allele (IVS-2-654 C-->T) by antisense RNA expression vector in cultured human erythroid cells.
Gong L; Gu XF; Chen YD; Ren ZR; Huang SZ; Zeng YT
Br J Haematol; 2000 Oct; 111(1):351-8. PubMed ID: 11091224
[TBL] [Abstract][Full Text] [Related]
27. CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia.
Zeng S; Lei S; Qu C; Wang Y; Teng S; Huang P
Hum Genet; 2023 Dec; 142(12):1677-1703. PubMed ID: 37878144
[TBL] [Abstract][Full Text] [Related]
28. Gene Therapy and Genome Editing.
Boulad F; Mansilla-Soto J; Cabriolu A; Rivière I; Sadelain M
Hematol Oncol Clin North Am; 2018 Apr; 32(2):329-342. PubMed ID: 29458735
[TBL] [Abstract][Full Text] [Related]
29. Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.
Thuret I; Ruggeri A; Angelucci E; Chabannon C
Stem Cells Transl Med; 2022 Apr; 11(4):407-414. PubMed ID: 35267028
[TBL] [Abstract][Full Text] [Related]
30. Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.
Phanthong P; Borwornpinyo S; Kitiyanant N; Jearawiriyapaisarn N; Nuntakarn L; Saetan J; Nualkaew T; Sa-Ngiamsuntorn K; Anurathapan U; Dinnyes A; Kitiyanant Y; Hongeng S
Stem Cells Transl Med; 2017 Apr; 6(4):1059-1069. PubMed ID: 28213976
[TBL] [Abstract][Full Text] [Related]
31. Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.
Persons DA
Hematology Am Soc Hematol Educ Program; 2009; ():690-7. PubMed ID: 20008255
[TBL] [Abstract][Full Text] [Related]
32. Cure for thalassemia major - from allogeneic hematopoietic stem cell transplantation to gene therapy.
Srivastava A; Shaji RV
Haematologica; 2017 Feb; 102(2):214-223. PubMed ID: 27909215
[TBL] [Abstract][Full Text] [Related]
33. Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.
Zhao HF; Abraham A; Kim YS; Wang YD; Pestina T; Zhan J; Humphries K; Nienhuis AW; Persons DA
Mol Ther; 2017 Mar; 25(3):593-605. PubMed ID: 28190779
[TBL] [Abstract][Full Text] [Related]
34. CRISPR-Cas9-mediated gene editing of the BCL11A enhancer for pediatric β
Fu B; Liao J; Chen S; Li W; Wang Q; Hu J; Yang F; Hsiao S; Jiang Y; Wang L; Chen F; Zhang Y; Wang X; Li D; Liu M; Wu Y
Nat Med; 2022 Aug; 28(8):1573-1580. PubMed ID: 35922667
[TBL] [Abstract][Full Text] [Related]
35. Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment.
Ali G; Tariq MA; Shahid K; Ahmad FJ; Akram J
Gene Ther; 2021 Feb; 28(1-2):6-15. PubMed ID: 32355226
[TBL] [Abstract][Full Text] [Related]
36. Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells.
Moiani A; Letort G; Lizot S; Chalumeau A; Foray C; Felix T; Le Clerre D; Temburni-Blake S; Hong P; Leduc S; Pinard N; Marechal A; Seclen E; Boyne A; Mayer L; Hong R; Pulicani S; Galetto R; Gouble A; Cavazzana M; Juillerat A; Miccio A; Duclert A; Duchateau P; Valton J
Nat Commun; 2024 Jun; 15(1):4965. PubMed ID: 38862518
[TBL] [Abstract][Full Text] [Related]
37. Recombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major.
Tian J; Wang F; Xue JF; Zhao F; Song LJ; Tan MQ
Int J Hematol; 2011 Jun; 93(6):691-699. PubMed ID: 21617888
[TBL] [Abstract][Full Text] [Related]
38. Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach.
Xie SY; Ren ZR; Zhang JZ; Guo XB; Wang QX; Wang S; Lin D; Gong XL; Li W; Huang SZ; Zeng F; Zeng YT
Hum Mol Genet; 2007 Nov; 16(21):2616-25. PubMed ID: 17716993
[TBL] [Abstract][Full Text] [Related]
39. 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]
40. CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation.
Gabr H; El Ghamrawy MK; Almaeen AH; Abdelhafiz AS; Hassan AOS; El Sissy MH
Stem Cell Res Ther; 2020 Sep; 11(1):390. PubMed ID: 32912325
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
