137 related articles for article (PubMed ID: 37989316)
1. 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]
2. 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]
3. 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]
4. Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.
Antoniou P; Hardouin G; Martinucci P; Frati G; Felix T; Chalumeau A; Fontana L; Martin J; Masson C; Brusson M; Maule G; Rosello M; Giovannangeli C; Abramowski V; de Villartay JP; Concordet JP; Del Bene F; El Nemer W; Amendola M; Cavazzana M; Cereseto A; Romano O; Miccio A
Nat Commun; 2022 Nov; 13(1):6618. PubMed ID: 36333351
[TBL] [Abstract][Full Text] [Related]
5. Therapeutic adenine base editing of human hematopoietic stem cells.
Liao J; Chen S; Hsiao S; Jiang Y; Yang Y; Zhang Y; Wang X; Lai Y; Bauer DE; Wu Y
Nat Commun; 2023 Jan; 14(1):207. PubMed ID: 36639729
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. 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]
8. 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]
9. 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]
10. 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]
11. 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]
12. 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]
13. 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]
14. 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]
15. Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34
Lamsfus-Calle A; Daniel-Moreno A; Antony JS; Epting T; Heumos L; Baskaran P; Admard J; Casadei N; Latifi N; Siegmund DM; Kormann MSD; Handgretinger R; Mezger M
Sci Rep; 2020 Jun; 10(1):10133. PubMed ID: 32576837
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. 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]
18. 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]
19. Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies.
Wang L; Li L; Ma Y; Hu H; Li Q; Yang Y; Liu W; Yin S; Li W; Fu B; Kurita R; Nakamura Y; Liu M; Lai Y; Li D
Cell Res; 2020 Mar; 30(3):276-278. PubMed ID: 31911671
[No Abstract] [Full Text] [Related]
20. Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies.
Wienert B; Martyn GE; Funnell APW; Quinlan KGR; Crossley M
Trends Genet; 2018 Dec; 34(12):927-940. PubMed ID: 30287096
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]