317 related articles for article (PubMed ID: 33620431)
1. In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice.
Li C; Georgakopoulou A; Mishra A; Gil S; Hawkins RD; Yannaki E; Lieber A
Blood Adv; 2021 Feb; 5(4):1122-1135. PubMed ID: 33620431
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies.
Li C; Georgakopoulou A; Newby GA; Everette KA; Nizamis E; Paschoudi K; Vlachaki E; Gil S; Anderson AK; Koob T; Huang L; Wang H; Kiem HP; Liu DR; Yannaki E; Lieber A
JCI Insight; 2022 Oct; 7(19):. PubMed ID: 36006707
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Featured Article: Modulation of fetal hemoglobin in hereditary persistence of fetal hemoglobin deletion type-2, compared to Sicilian δβ-thalassemia, by BCL11A and SOX6-targeting microRNAs.
Fornari TA; Lanaro C; Albuquerque DM; Ferreira R; Costa FF
Exp Biol Med (Maywood); 2017 Feb; 242(3):267-274. PubMed ID: 27591578
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Original Research: Generation of non-deletional hereditary persistence of fetal hemoglobin β-globin locus yeast artificial chromosome transgenic mouse models: -175 Black HPFH and -195 Brazilian HPFH.
Braghini CA; Costa FC; Fedosyuk H; Neades RY; Novikova LV; Parker MP; Winefield RD; Peterson KR
Exp Biol Med (Maywood); 2016 Apr; 241(7):697-705. PubMed ID: 26946532
[TBL] [Abstract][Full Text] [Related]
8. 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]
9. 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]
10. Base editing of key residues in the BCL11A-XL-specific zinc finger domains derepresses fetal globin expression.
Rajendiran V; Devaraju N; Haddad M; Ravi NS; Panigrahi L; Paul J; Gopalakrishnan C; Wyman S; Ariudainambi K; Mahalingam G; Periyasami Y; Prasad K; George A; Sukumaran D; Gopinathan S; Pai AA; Nakamura Y; Balasubramanian P; Ramalingam R; Thangavel S; Velayudhan SR; Corn JE; Mackay JP; Marepally S; Srivastava A; Crossley M; Mohankumar KM
Mol Ther; 2024 Mar; 32(3):663-677. PubMed ID: 38273654
[TBL] [Abstract][Full Text] [Related]
11. A natural regulatory mutation in the proximal promoter elevates fetal
Martyn GE; Wienert B; Kurita R; Nakamura Y; Quinlan KGR; Crossley M
Blood; 2019 Feb; 133(8):852-856. PubMed ID: 30617196
[TBL] [Abstract][Full Text] [Related]
12. Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs.
Han W; Qiu HY; Sun S; Fu ZC; Wang GQ; Qian X; Wang L; Zhai X; Wei J; Wang Y; Guo YL; Cao GH; Ji RJ; Zhang YZ; Ma H; Wang H; Zhao M; Wu J; Bi L; Chen QB; Li Z; Yu L; Mou X; Yin H; Yang L; Chen J; Yang B; Zhang Y
Cell Stem Cell; 2023 Dec; 30(12):1624-1639.e8. PubMed ID: 37989316
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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]
15. Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding.
Martyn GE; Wienert B; Yang L; Shah M; Norton LJ; Burdach J; Kurita R; Nakamura Y; Pearson RCM; Funnell APW; Quinlan KGR; Crossley M
Nat Genet; 2018 Apr; 50(4):498-503. PubMed ID: 29610478
[TBL] [Abstract][Full Text] [Related]
16. In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model.
Li C; Wang H; Georgakopoulou A; Gil S; Yannaki E; Lieber A
Mol Ther; 2021 Feb; 29(2):822-837. PubMed ID: 32949495
[TBL] [Abstract][Full Text] [Related]
17. ATF4 Regulates MYB to Increase γ-Globin in Response to Loss of β-Globin.
Boontanrart MY; Schröder MS; Stehli GM; Banović M; Wyman SK; Lew RJ; Bordi M; Gowen BG; DeWitt MA; Corn JE
Cell Rep; 2020 Aug; 32(5):107993. PubMed ID: 32755585
[TBL] [Abstract][Full Text] [Related]
18. Triplex-forming peptide nucleic acids induce heritable elevations in gamma-globin expression in hematopoietic progenitor cells.
Chin JY; Reza F; Glazer PM
Mol Ther; 2013 Mar; 21(3):580-7. PubMed ID: 23337982
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies.
Paschoudi K; Yannaki E; Psatha N
Int J Mol Sci; 2023 May; 24(11):. PubMed ID: 37298481
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
