319 related articles for article (PubMed ID: 28344999)
1. 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]
2. Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major.
Psatha N; Reik A; Phelps S; Zhou Y; Dalas D; Yannaki E; Levasseur DN; Urnov FD; Holmes MC; Papayannopoulou T
Mol Ther Methods Clin Dev; 2018 Sep; 10():313-326. PubMed ID: 30182035
[TBL] [Abstract][Full Text] [Related]
3. BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity.
Demirci S; Zeng J; Wu Y; Uchida N; Shen AH; Pellin D; Gamer J; Yapundich M; Drysdale C; Bonanno J; Bonifacino AC; Krouse AE; Linde NS; Engels T; Donahue RE; Haro-Mora JJ; Leonard A; Nassehi T; Luk K; Porter SN; Lazzarotto CR; Tsai SQ; Weiss MJ; Pruett-Miller SM; Wolfe SA; Bauer DE; Tisdale JF
J Clin Invest; 2020 Dec; 130(12):6677-6687. PubMed ID: 32897878
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models.
Demirci S; Khan MBN; Hinojosa G; Le A; Leonard A; Essawi K; Gudmundsdottir B; Liu X; Zeng J; Inam Z; Chu R; Uchida N; Araki D; London E; Butt H; Maitland SA; Bauer DE; Wolfe SA; Larochelle A; Tisdale JF
Cytotherapy; 2024 Jun; 26(6):641-648. PubMed ID: 38506770
[TBL] [Abstract][Full Text] [Related]
6. Detection of CRISPR/Cas9-Mediated Fetal Hemoglobin Reactivation in Erythroblasts Derived from Cord Blood-Hematopoietic Stem Cells.
Maroofi N; Maleki MSM; Tahmasebi M; Khorshid HRK; Modaberi Y; Najafipour R; Banan M
Mol Biotechnol; 2024 Apr; ():. PubMed ID: 38649638
[TBL] [Abstract][Full Text] [Related]
7. 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]
8. 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]
9. 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]
10. 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]
11. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H; Altshuler D; Cappellini MD; Chen YS; Domm J; Eustace BK; Foell J; de la Fuente J; Grupp S; Handgretinger R; Ho TW; Kattamis A; Kernytsky A; Lekstrom-Himes J; Li AM; Locatelli F; Mapara MY; de Montalembert M; Rondelli D; Sharma A; Sheth S; Soni S; Steinberg MH; Wall D; Yen A; Corbacioglu S
N Engl J Med; 2021 Jan; 384(3):252-260. PubMed ID: 33283989
[TBL] [Abstract][Full Text] [Related]
12. Inhibition of γ/β Globin Gene Switching in CD 34
Taghavi SA; Hosseini KM; Tamaddon G; Kasraian L
Indian J Hematol Blood Transfus; 2019 Oct; 35(4):758-764. PubMed ID: 31741633
[TBL] [Abstract][Full Text] [Related]
13. 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]
14. CRISPR-Cas9 Editing of the
Sharma A; Boelens JJ; Cancio M; Hankins JS; Bhad P; Azizy M; Lewandowski A; Zhao X; Chitnis S; Peddinti R; Zheng Y; Kapoor N; Ciceri F; Maclachlan T; Yang Y; Liu Y; Yuan J; Naumann U; Yu VWC; Stevenson SC; De Vita S; LaBelle JL
N Engl J Med; 2023 Aug; 389(9):820-832. PubMed ID: 37646679
[TBL] [Abstract][Full Text] [Related]
15. 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]
16. Gene editing without
Zeng J; Nguyen MA; Liu P; Ferreira da Silva L; Lin LY; Justus DG; Petri K; Clement K; Porter SN; Verma A; Neri NR; Rosanwo T; Ciuculescu MF; Abriss D; Mintzer E; Maitland SA; Demirci S; Tisdale JF; Williams DA; Zhu LJ; Pruett-Miller SM; Pinello L; Joung JK; Pattanayak V; Manis JP; Armant M; Pellin D; Brendel C; Wolfe SA; Bauer DE
bioRxiv; 2023 May; ():. PubMed ID: 37292647
[TBL] [Abstract][Full Text] [Related]
17. Expression analysis data of BCL11A and γ-globin genes in KU812 and KG-1 cell lines after CRISPR/Cas9-mediated BCL11A enhancer deletion.
Khosravi MA; Abbasalipour M; Concordet JP; Berg JV; Zeinali S; Arashkia A; Buch T; Karimipoor M
Data Brief; 2020 Feb; 28():104974. PubMed ID: 31890812
[TBL] [Abstract][Full Text] [Related]
18. Sustained fetal hemoglobin induction in vivo is achieved by
Uchida N; Ferrara F; Drysdale CM; Yapundich M; Gamer J; Nassehi T; DiNicola J; Shibata Y; Wielgosz M; Kim YS; Bauler M; Throm RE; Haro-Mora JJ; Demirci S; Bonifacino AC; Krouse AE; Linde NS; Donahue RE; Ryu B; Tisdale JF
Sci Transl Med; 2021 Apr; 13(591):. PubMed ID: 33910976
[TBL] [Abstract][Full Text] [Related]
19. Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.
Bjurström CF; Mojadidi M; Phillips J; Kuo C; Lai S; Lill GR; Cooper A; Kaufman M; Urbinati F; Wang X; Hollis RP; Kohn DB
Mol Ther Nucleic Acids; 2016; 5(8):e351. PubMed ID: 28131278
[TBL] [Abstract][Full Text] [Related]
20. Strict in vivo specificity of the
Smith EC; Luc S; Croney DM; Woodworth MB; Greig LC; Fujiwara Y; Nguyen M; Sher F; Macklis JD; Bauer DE; Orkin SH
Blood; 2016 Nov; 128(19):2338-2342. PubMed ID: 27707736
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
