These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

318 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. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. 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]  

  • 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]
    of 16.