383 related articles for article (PubMed ID: 27599293)
1. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
Brendel C; Guda S; Renella R; Bauer DE; Canver MC; Kim YJ; Heeney MM; Klatt D; Fogel J; Milsom MD; Orkin SH; Gregory RI; Williams DA
J Clin Invest; 2016 Oct; 126(10):3868-3878. PubMed ID: 27599293
[TBL] [Abstract][Full Text] [Related]
2. 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]
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. 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]
5. 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]
6. 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]
7. 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]
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. 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]
10. 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]
11. 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]
12. 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]
13. 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]
14. 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]
15. 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]
16. Long-Term Engraftment and Fetal Globin Induction upon
Chang KH; Smith SE; Sullivan T; Chen K; Zhou Q; West JA; Liu M; Liu Y; Vieira BF; Sun C; Hong VP; Zhang M; Yang X; Reik A; Urnov FD; Rebar EJ; Holmes MC; Danos O; Jiang H; Tan S
Mol Ther Methods Clin Dev; 2017 Mar; 4():137-148. PubMed ID: 28344999
[TBL] [Abstract][Full Text] [Related]
17. 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]
18. 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]
19. Expression of an anti-sickling beta-globin in human erythroblasts derived from retrovirally transduced primitive normal and sickle cell disease hematopoietic cells.
Oh IH; Fabry ME; Humphries RK; Pawliuk R; Leboulch P; Hoffman R; Nagel RL; Eaves C
Exp Hematol; 2004 May; 32(5):461-9. PubMed ID: 15145214
[TBL] [Abstract][Full Text] [Related]
20. Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Park SH; Lee CM; Dever DP; Davis TH; Camarena J; Srifa W; Zhang Y; Paikari A; Chang AK; Porteus MH; Sheehan VA; Bao G
Nucleic Acids Res; 2019 Sep; 47(15):7955-7972. PubMed ID: 31147717
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]