1060 related articles for article (PubMed ID: 27820943)
41. Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.
Rosanwo TO; Bauer DE
Mol Ther; 2021 Nov; 29(11):3163-3178. PubMed ID: 34628053
[TBL] [Abstract][Full Text] [Related]
42. Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia.
Mettananda S; Fisher CA; Hay D; Badat M; Quek L; Clark K; Hublitz P; Downes D; Kerry J; Gosden M; Telenius J; Sloane-Stanley JA; Faustino P; Coelho A; Doondeea J; Usukhbayar B; Sopp P; Sharpe JA; Hughes JR; Vyas P; Gibbons RJ; Higgs DR
Nat Commun; 2017 Sep; 8(1):424. PubMed ID: 28871148
[TBL] [Abstract][Full Text] [Related]
43. CRISPR/Cas9-mediated β-globin gene knockout in rabbits recapitulates human β-thalassemia.
Yang Y; Kang X; Hu S; Chen B; Xie Y; Song B; Zhang Q; Wu H; Ou Z; Xian Y; Fan Y; Li X; Lai L; Sun X
J Biol Chem; 2021; 296():100464. PubMed ID: 33639162
[TBL] [Abstract][Full Text] [Related]
44. 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]
45. Genetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system.
Trakarnsanga K; Thongsin N; Metheetrairut C; Tipgomut C; Poldee S; Wattanapanitch M
Sci Rep; 2022 Sep; 12(1):15551. PubMed ID: 36114353
[TBL] [Abstract][Full Text] [Related]
46. Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates.
Lamsfus-Calle A; Daniel-Moreno A; Ureña-Bailén G; Rottenberger J; Raju J; Epting T; Marciano S; Heumos L; Baskaran P; S Antony J; Handgretinger R; Mezger M
CRISPR J; 2021 Apr; 4(2):207-222. PubMed ID: 33876951
[TBL] [Abstract][Full Text] [Related]
47. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
Urbinati F; Campo Fernandez B; Masiuk KE; Poletti V; Hollis RP; Koziol C; Kaufman ML; Brown D; Mavilio F; Kohn DB
Hum Gene Ther; 2018 Oct; 29(10):1153-1166. PubMed ID: 30198339
[TBL] [Abstract][Full Text] [Related]
48. CRISPR/Cas9 genome editing in human hematopoietic stem cells.
Bak RO; Dever DP; Porteus MH
Nat Protoc; 2018 Feb; 13(2):358-376. PubMed ID: 29370156
[TBL] [Abstract][Full Text] [Related]
49. NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia.
Román-Rodríguez FJ; Ugalde L; Álvarez L; Díez B; Ramírez MJ; Risueño C; Cortón M; Bogliolo M; Bernal S; March F; Ayuso C; Hanenberg H; Sevilla J; Rodríguez-Perales S; Torres-Ruiz R; Surrallés J; Bueren JA; Río P
Cell Stem Cell; 2019 Nov; 25(5):607-621.e7. PubMed ID: 31543367
[TBL] [Abstract][Full Text] [Related]
50. CRISPR/Cas9 gene editing for curing sickle cell disease.
Park SH; Bao G
Transfus Apher Sci; 2021 Feb; 60(1):103060. PubMed ID: 33455878
[TBL] [Abstract][Full Text] [Related]
51. Gene Therapy for β-Hemoglobinopathies.
Cavazzana M; Antoniani C; Miccio A
Mol Ther; 2017 May; 25(5):1142-1154. PubMed ID: 28377044
[TBL] [Abstract][Full Text] [Related]
52. 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]
53. 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]
54. Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia.
Ma SP; Gao XX; Zhou GQ; Zhang HK; Yang JM; Wang WJ; Song XM; Chen HY; Lu DR
Gene; 2022 Apr; 820():146289. PubMed ID: 35143940
[TBL] [Abstract][Full Text] [Related]
55. Engraftment of immune-deficient mice with primitive hematopoietic cells from beta-thalassemia and sickle cell anemia patients: implications for evaluating human gene therapy protocols.
Larochelle A; Vormoor J; Lapidot T; Sher G; Furukawa T; Li Q; Shultz LD; Olivieri NF; Stamatoyannopoulos G; Dick JE
Hum Mol Genet; 1995 Feb; 4(2):163-72. PubMed ID: 7757063
[TBL] [Abstract][Full Text] [Related]
56. Machine learning-based approaches for identifying human blood cells harboring CRISPR-mediated fetal chromatin domain ablations.
Li Y; Zaheri S; Nguyen K; Liu L; Hassanipour F; Pace BS; Bleris L
Sci Rep; 2022 Jan; 12(1):1481. PubMed ID: 35087158
[TBL] [Abstract][Full Text] [Related]
57. Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Patsali P; Turchiano G; Papasavva P; Romito M; Loucari CC; Stephanou C; Christou S; Sitarou M; Mussolino C; Cornu TI; Antoniou MN; Lederer CW; Cathomen T; Kleanthous M
Haematologica; 2019 Nov; 104(11):e497-e501. PubMed ID: 31004018
[No Abstract] [Full Text] [Related]
58. Gene therapy using haematopoietic stem and progenitor cells.
Ferrari G; Thrasher AJ; Aiuti A
Nat Rev Genet; 2021 Apr; 22(4):216-234. PubMed ID: 33303992
[TBL] [Abstract][Full Text] [Related]
59. 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]
60. Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
Breda L; Casu C; Gardenghi S; Bianchi N; Cartegni L; Narla M; Yazdanbakhsh K; Musso M; Manwani D; Little J; Gardner LB; Kleinert DA; Prus E; Fibach E; Grady RW; Giardina PJ; Gambari R; Rivella S
PLoS One; 2012; 7(3):e32345. PubMed ID: 22479321
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
