BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

708 related articles for article (PubMed ID: 30424953)

  • 1. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.
    Lattanzi A; Meneghini V; Pavani G; Amor F; Ramadier S; Felix T; Antoniani C; Masson C; Alibeu O; Lee C; Porteus MH; Bao G; Amendola M; Mavilio F; Miccio A
    Mol Ther; 2019 Jan; 27(1):137-150. PubMed ID: 30424953
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia.
    Ye L; Wang J; Tan Y; Beyer AI; Xie F; Muench MO; Kan YW
    Proc Natl Acad Sci U S A; 2016 Sep; 113(38):10661-5. PubMed ID: 27601644
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. TRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem Cells.
    Yen J; Fiorino M; Liu Y; Paula S; Clarkson S; Quinn L; Tschantz WR; Klock H; Guo N; Russ C; Yu VWC; Mickanin C; Stevenson SC; Lee C; Yang Y
    Sci Rep; 2018 Nov; 8(1):16304. PubMed ID: 30389991
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies.
    Paschoudi K; Yannaki E; Psatha N
    Int J Mol Sci; 2023 May; 24(11):. PubMed ID: 37298481
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. 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]  

  • 11. 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]  

  • 12. Chemical Modification of Guide RNAs for Improved CRISPR Activity in CD34+ Human Hematopoietic Stem and Progenitor Cells.
    Shapiro J; Tovin A; Iancu O; Allen D; Hendel A
    Methods Mol Biol; 2021; 2162():37-48. PubMed ID: 32926376
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration.
    Bloomer H; Smith RH; Hakami W; Larochelle A
    Mol Ther; 2021 Apr; 29(4):1611-1624. PubMed ID: 33309880
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Induction of therapeutic levels of HbF in genome-edited primary β
    Mingoia M; Caria CA; Ye L; Asunis I; Marongiu MF; Manunza L; Sollaino MC; Wang J; Cabriolu A; Kurita R; Nakamura Y; Cucca F; Kan YW; Marini MG; Moi P
    Br J Haematol; 2021 Jan; 192(2):395-404. PubMed ID: 33216968
    [TBL] [Abstract][Full Text] [Related]  

  • 16. CRISPR/Cas9 system and its applications in human hematopoietic cells.
    Hu X
    Blood Cells Mol Dis; 2016 Nov; 62():6-12. PubMed ID: 27736664
    [TBL] [Abstract][Full Text] [Related]  

  • 17. 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]  

  • 18. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.
    DeWitt MA; Magis W; Bray NL; Wang T; Berman JR; Urbinati F; Heo SJ; Mitros T; Muñoz DP; Boffelli D; Kohn DB; Walters MC; Carroll D; Martin DI; Corn JE
    Sci Transl Med; 2016 Oct; 8(360):360ra134. PubMed ID: 27733558
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20. 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]  

    [Next]    [New Search]
    of 36.