These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

562 related articles for article (PubMed ID: 29789357)

  • 1. 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]  

  • 2. 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]  

  • 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. 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]  

  • 5. 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]  

  • 6. In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model.
    Li C; Wang H; Georgakopoulou A; Gil S; Yannaki E; Lieber A
    Mol Ther; 2021 Feb; 29(2):822-837. PubMed ID: 32949495
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells.
    Pavani G; Fabiano A; Laurent M; Amor F; Cantelli E; Chalumeau A; Maule G; Tachtsidi A; Concordet JP; Cereseto A; Mavilio F; Ferrari G; Miccio A; Amendola M
    Blood Adv; 2021 Mar; 5(5):1137-1153. PubMed ID: 33635334
    [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. Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors.
    Li C; Mishra AS; Gil S; Wang M; Georgakopoulou A; Papayannopoulou T; Hawkins RD; Lieber A
    Mol Ther; 2019 Dec; 27(12):2195-2212. PubMed ID: 31494053
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements.
    Wang H; Georgakopoulou A; Li C; Liu Z; Gil S; Bashyam A; Yannaki E; Anagnostopoulos A; Pande A; Izsvák Z; Papayannopoulou T; Lieber A
    JCI Insight; 2020 Aug; 5(16):. PubMed ID: 32814708
    [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. 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]  

  • 14. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
    Antoniani C; Meneghini V; Lattanzi A; Felix T; Romano O; Magrin E; Weber L; Pavani G; El Hoss S; Kurita R; Nakamura Y; Cradick TJ; Lundberg AS; Porteus M; Amendola M; El Nemer W; Cavazzana M; Mavilio F; Miccio A
    Blood; 2018 Apr; 131(17):1960-1973. PubMed ID: 29519807
    [TBL] [Abstract][Full Text] [Related]  

  • 15. The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice.
    Ou Z; Niu X; He W; Chen Y; Song B; Xian Y; Fan D; Tang D; Sun X
    Sci Rep; 2016 Sep; 6():32463. PubMed ID: 27581487
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia.
    Bao X; Zhang X; Wang L; Wang Z; Huang J; Zhang Q; Ye Y; Liu Y; Chen D; Zuo Y; Liu Q; Xu P; Huang B; Fang J; Lao J; Feng X; Li Y; Kurita R; Nakamura Y; Yu W; Ju C; Huang C; Mohandas N; Li D; Zhao C; Xu X
    Am J Hum Genet; 2021 Apr; 108(4):709-721. PubMed ID: 33735615
    [TBL] [Abstract][Full Text] [Related]  

  • 17. CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.
    Dever DP; Bak RO; Reinisch A; Camarena J; Washington G; Nicolas CE; Pavel-Dinu M; Saxena N; Wilkens AB; Mantri S; Uchida N; Hendel A; Narla A; Majeti R; Weinberg KI; Porteus MH
    Nature; 2016 Nov; 539(7629):384-389. PubMed ID: 27820943
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

  • 19. 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]  

  • 20. 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]  

    [Next]    [New Search]
    of 29.