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8. 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]
9. 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]
10. 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]
11. 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]
12. 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]
13. 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]
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15. 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]
16. MicroRNA-486-3p regulates γ-globin expression in human erythroid cells by directly modulating BCL11A. Lulli V; Romania P; Morsilli O; Cianciulli P; Gabbianelli M; Testa U; Giuliani A; Marziali G PLoS One; 2013; 8(4):e60436. PubMed ID: 23593217 [TBL] [Abstract][Full Text] [Related]
17. 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]