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2. Transcriptional Repressor BCL11A in Erythroid Cells. Zheng G; Orkin SH Adv Exp Med Biol; 2024; 1459():199-215. PubMed ID: 39017845 [TBL] [Abstract][Full Text] [Related]
3. Post-Transcriptional Genetic Silencing of Esrick EB; Lehmann LE; Biffi A; Achebe M; Brendel C; Ciuculescu MF; Daley H; MacKinnon B; Morris E; Federico A; Abriss D; Boardman K; Khelladi R; Shaw K; Negre H; Negre O; Nikiforow S; Ritz J; Pai SY; London WB; Dansereau C; Heeney MM; Armant M; Manis JP; Williams DA N Engl J Med; 2021 Jan; 384(3):205-215. PubMed ID: 33283990 [TBL] [Abstract][Full Text] [Related]
4. Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies. Liu B; Brendel C; Vinjamur DS; Zhou Y; Harris C; McGuinness M; Manis JP; Bauer DE; Xu H; Williams DA Mol Ther; 2022 Aug; 30(8):2693-2708. PubMed ID: 35526095 [TBL] [Abstract][Full Text] [Related]
6. 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]
7. Bcl11a Deficiency Leads to Hematopoietic Stem Cell Defects with an Aging-like Phenotype. Luc S; Huang J; McEldoon JL; Somuncular E; Li D; Rhodes C; Mamoor S; Hou S; Xu J; Orkin SH Cell Rep; 2016 Sep; 16(12):3181-3194. PubMed ID: 27653684 [TBL] [Abstract][Full Text] [Related]
8. Single-cell transcriptomic reconstruction reveals cell cycle and multi-lineage differentiation defects in Bcl11a-deficient hematopoietic stem cells. Tsang JC; Yu Y; Burke S; Buettner F; Wang C; Kolodziejczyk AA; Teichmann SA; Lu L; Liu P Genome Biol; 2015 Sep; 16():178. PubMed ID: 26387834 [TBL] [Abstract][Full Text] [Related]
9. 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]
10. Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A. Sankaran VG; Menne TF; Xu J; Akie TE; Lettre G; Van Handel B; Mikkola HK; Hirschhorn JN; Cantor AB; Orkin SH Science; 2008 Dec; 322(5909):1839-42. PubMed ID: 19056937 [TBL] [Abstract][Full Text] [Related]
11. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34 Katta V; O'Keefe K; Li Y; Mayuranathan T; Lazzarotto CR; Wood RK; Levine RM; Powers A; Mayberry K; Manquen G; Yao Y; Zhang J; Jang Y; Nimmagadda N; Dempsey EA; Lee G; Uchida N; Cheng Y; Fazio F; Lockey T; Meagher M; Sharma A; Tisdale JF; Zhou S; Yen JS; Weiss MJ; Tsai SQ Mol Ther; 2024 Oct; 32(10):3433-3452. PubMed ID: 39086133 [TBL] [Abstract][Full Text] [Related]
12. miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction. Guda S; Brendel C; Renella R; Du P; Bauer DE; Canver MC; Grenier JK; Grimson AW; Kamran SC; Thornton J; de Boer H; Root DE; Milsom MD; Orkin SH; Gregory RI; Williams DA Mol Ther; 2015 Sep; 23(9):1465-74. PubMed ID: 26080908 [TBL] [Abstract][Full Text] [Related]
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14. 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]
15. Engraftment of immune-deficient mice with primitive hematopoietic cells from beta-thalassemia and sickle cell anemia patients: implications for evaluating human gene therapy protocols. Larochelle A; Vormoor J; Lapidot T; Sher G; Furukawa T; Li Q; Shultz LD; Olivieri NF; Stamatoyannopoulos G; Dick JE Hum Mol Genet; 1995 Feb; 4(2):163-72. PubMed ID: 7757063 [TBL] [Abstract][Full Text] [Related]
16. Transcriptional regulators Myb and BCL11A interplay with DNA methyltransferase 1 in developmental silencing of embryonic and fetal β-like globin genes. Roosjen M; McColl B; Kao B; Gearing LJ; Blewitt ME; Vadolas J FASEB J; 2014 Apr; 28(4):1610-20. PubMed ID: 24371119 [TBL] [Abstract][Full Text] [Related]
17. 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]
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19. POGZ Is Required for Silencing Mouse Embryonic β-like Hemoglobin and Human Fetal Hemoglobin Expression. Gudmundsdottir B; Gudmundsson KO; Klarmann KD; Singh SK; Sun L; Singh S; Du Y; Coppola V; Stockwin L; Nguyen N; Tessarollo L; Thorsteinsson L; Sigurjonsson OE; Gudmundsson S; Rafnar T; Tisdale JF; Keller JR Cell Rep; 2018 Jun; 23(11):3236-3248. PubMed ID: 29898395 [TBL] [Abstract][Full Text] [Related]
20. 2p15-p16.1 microdeletions encompassing and proximal to BCL11A are associated with elevated HbF in addition to neurologic impairment. Funnell AP; Prontera P; Ottaviani V; Piccione M; Giambona A; Maggio A; Ciaffoni F; Stehling-Sun S; Marra M; Masiello F; Varricchio L; Stamatoyannopoulos JA; Migliaccio AR; Papayannopoulou T Blood; 2015 Jul; 126(1):89-93. PubMed ID: 26019277 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]