173 related articles for article (PubMed ID: 35143940)
1. 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]
2. 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]
3. Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: A promising approach for gene therapy of beta thalassemia disease.
Khosravi MA; Abbasalipour M; Concordet JP; Berg JV; Zeinali S; Arashkia A; Azadmanesh K; Buch T; Karimipoor M
Eur J Pharmacol; 2019 Jul; 854():398-405. PubMed ID: 31039344
[TBL] [Abstract][Full Text] [Related]
4. Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing.
Li C; Psatha N; Sova P; Gil S; Wang H; Kim J; Kulkarni C; Valensisi C; Hawkins RD; Stamatoyannopoulos G; Lieber A
Blood; 2018 Jun; 131(26):2915-2928. PubMed ID: 29789357
[TBL] [Abstract][Full Text] [Related]
5. CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression.
Han Y; Tan X; Jin T; Zhao S; Hu L; Zhang W; Kurita R; Nakamura Y; Liu J; Li D; Zhang Z; Fang X; Huang S
Eur J Pharmacol; 2022 Mar; 918():174788. PubMed ID: 35093321
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia.
Bao X; Zhang X; Wang L; Wang Z; Huang J; Zhang Q; Ye Y; Liu Y; Chen D; Zuo Y; Liu Q; Xu P; Huang B; Fang J; Lao J; Feng X; Li Y; Kurita R; Nakamura Y; Yu W; Ju C; Huang C; Mohandas N; Li D; Zhao C; Xu X
Am J Hum Genet; 2021 Apr; 108(4):709-721. PubMed ID: 33735615
[TBL] [Abstract][Full Text] [Related]
8. Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Wu Y; Zeng J; Roscoe BP; Liu P; Yao Q; Lazzarotto CR; Clement K; Cole MA; Luk K; Baricordi C; Shen AH; Ren C; Esrick EB; Manis JP; Dorfman DM; Williams DA; Biffi A; Brugnara C; Biasco L; Brendel C; Pinello L; Tsai SQ; Wolfe SA; Bauer DE
Nat Med; 2019 May; 25(5):776-783. PubMed ID: 30911135
[TBL] [Abstract][Full Text] [Related]
9. The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.
Papanikolaou E; Georgomanoli M; Stamateris E; Panetsos F; Karagiorga M; Tsaftaridis P; Graphakos S; Anagnou NP
Hum Gene Ther; 2012 Jan; 23(1):15-31. PubMed ID: 21875313
[TBL] [Abstract][Full Text] [Related]
10. Disruption of SOX6 gene using CRISPR/Cas9 technology for gamma-globin reactivation: An approach towards gene therapy of β-thalassemia.
Shariati L; Rohani F; Heidari Hafshejani N; Kouhpayeh S; Boshtam M; Mirian M; Rahimmanesh I; Hejazi Z; Modarres M; Pieper IL; Khanahmad H
J Cell Biochem; 2018 Nov; 119(11):9357-9363. PubMed ID: 30010219
[TBL] [Abstract][Full Text] [Related]
11. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells.
Chung JE; Magis W; Vu J; Heo SJ; Wartiovaara K; Walters MC; Kurita R; Nakamura Y; Boffelli D; Martin DIK; Corn JE; DeWitt MA
PLoS One; 2019; 14(1):e0208237. PubMed ID: 30645582
[TBL] [Abstract][Full Text] [Related]
12. The Novel Role of the B-Cell Lymphoma/Leukemia 11A (BCL11A) Gene in β-Thalassaemia Treatment.
Mahmoud Ahmed NH; Lai MI
Cardiovasc Hematol Disord Drug Targets; 2023; 22(4):226-236. PubMed ID: 36734897
[TBL] [Abstract][Full Text] [Related]
13. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia.
Ye L; Wang J; Tan Y; Beyer AI; Xie F; Muench MO; Kan YW
Proc Natl Acad Sci U S A; 2016 Sep; 113(38):10661-5. PubMed ID: 27601644
[TBL] [Abstract][Full Text] [Related]
14. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Métais JY; Doerfler PA; Mayuranathan T; Bauer DE; Fowler SC; Hsieh MM; Katta V; Keriwala S; Lazzarotto CR; Luk K; Neel MD; Perry SS; Peters ST; Porter SN; Ryu BY; Sharma A; Shea D; Tisdale JF; Uchida N; Wolfe SA; Woodard KJ; Wu Y; Yao Y; Zeng J; Pruett-Miller S; Tsai SQ; Weiss MJ
Blood Adv; 2019 Nov; 3(21):3379-3392. PubMed ID: 31698466
[TBL] [Abstract][Full Text] [Related]
15. Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin.
Ravi NS; Wienert B; Wyman SK; Bell HW; George A; Mahalingam G; Vu JT; Prasad K; Bandlamudi BP; Devaraju N; Rajendiran V; Syedbasha N; Pai AA; Nakamura Y; Kurita R; Narayanasamy M; Balasubramanian P; Thangavel S; Marepally S; Velayudhan SR; Srivastava A; DeWitt MA; Crossley M; Corn JE; Mohankumar KM
Elife; 2022 Feb; 11():. PubMed ID: 35147495
[TBL] [Abstract][Full Text] [Related]
16. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
Weber L; Frati G; Felix T; Hardouin G; Casini A; Wollenschlaeger C; Meneghini V; Masson C; De Cian A; Chalumeau A; Mavilio F; Amendola M; Andre-Schmutz I; Cereseto A; El Nemer W; Concordet JP; Giovannangeli C; Cavazzana M; Miccio A
Sci Adv; 2020 Feb; 6(7):. PubMed ID: 32917636
[TBL] [Abstract][Full Text] [Related]
17. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H; Altshuler D; Cappellini MD; Chen YS; Domm J; Eustace BK; Foell J; de la Fuente J; Grupp S; Handgretinger R; Ho TW; Kattamis A; Kernytsky A; Lekstrom-Himes J; Li AM; Locatelli F; Mapara MY; de Montalembert M; Rondelli D; Sharma A; Sheth S; Soni S; Steinberg MH; Wall D; Yen A; Corbacioglu S
N Engl J Med; 2021 Jan; 384(3):252-260. PubMed ID: 33283989
[TBL] [Abstract][Full Text] [Related]
18. A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
Traxler EA; Yao Y; Wang YD; Woodard KJ; Kurita R; Nakamura Y; Hughes JR; Hardison RC; Blobel GA; Li C; Weiss MJ
Nat Med; 2016 Sep; 22(9):987-90. PubMed ID: 27525524
[TBL] [Abstract][Full Text] [Related]
19. The HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression.
Huang P; Peslak SA; Lan X; Khandros E; Yano JA; Sharma M; Keller CA; Giardine B; Qin K; Abdulmalik O; Hardison RC; Shi J; Blobel GA
Blood; 2020 Jun; 135(24):2121-2132. PubMed ID: 32299090
[TBL] [Abstract][Full Text] [Related]
20. Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene.
Zhao H; Pestina TI; Nasimuzzaman M; Mehta P; Hargrove PW; Persons DA
Blood; 2009 Jun; 113(23):5747-56. PubMed ID: 19365082
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]