BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

596 related articles for article (PubMed ID: 31178391)

  • 1. Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.
    Romero Z; Lomova A; Said S; Miggelbrink A; Kuo CY; Campo-Fernandez B; Hoban MD; Masiuk KE; Clark DN; Long J; Sanchez JM; Velez M; Miyahira E; Zhang R; Brown D; Wang X; Kurmangaliyev YZ; Hollis RP; Kohn DB
    Mol Ther; 2019 Aug; 27(8):1389-1406. PubMed ID: 31178391
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
    Newby GA; Yen JS; Woodard KJ; Mayuranathan T; Lazzarotto CR; Li Y; Sheppard-Tillman H; Porter SN; Yao Y; Mayberry K; Everette KA; Jang Y; Podracky CJ; Thaman E; Lechauve C; Sharma A; Henderson JM; Richter MF; Zhao KT; Miller SM; Wang T; Koblan LW; McCaffrey AP; Tisdale JF; Kalfa TA; Pruett-Miller SM; Tsai SQ; Weiss MJ; Liu DR
    Nature; 2021 Jul; 595(7866):295-302. PubMed ID: 34079130
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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]  

  • 5. CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
    Hoban MD; Lumaquin D; Kuo CY; Romero Z; Long J; Ho M; Young CS; Mojadidi M; Fitz-Gibbon S; Cooper AR; Lill GR; Urbinati F; Campo-Fernandez B; Bjurstrom CF; Pellegrini M; Hollis RP; Kohn DB
    Mol Ther; 2016 Sep; 24(9):1561-9. PubMed ID: 27406980
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
    Lattanzi A; Camarena J; Lahiri P; Segal H; Srifa W; Vakulskas CA; Frock RL; Kenrick J; Lee C; Talbott N; Skowronski J; Cromer MK; Charlesworth CT; Bak RO; Mantri S; Bao G; DiGiusto D; Tisdale J; Wright JF; Bhatia N; Roncarolo MG; Dever DP; Porteus MH
    Sci Transl Med; 2021 Jun; 13(598):. PubMed ID: 34135108
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.
    Wilkinson AC; Dever DP; Baik R; Camarena J; Hsu I; Charlesworth CT; Morita C; Nakauchi H; Porteus MH
    Nat Commun; 2021 Jan; 12(1):686. PubMed ID: 33514718
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Development of gene editing strategies for human β-globin (HBB) gene mutations.
    Kalkan BM; Kala EY; Yuce M; Karadag Alpaslan M; Kocabas F
    Gene; 2020 Apr; 734():144398. PubMed ID: 31987908
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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]  

  • 10. A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.
    Cai L; Bai H; Mahairaki V; Gao Y; He C; Wen Y; Jin YC; Wang Y; Pan RL; Qasba A; Ye Z; Cheng L
    Stem Cells Transl Med; 2018 Jan; 7(1):87-97. PubMed ID: 29164808
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
    Hoban MD; Cost GJ; Mendel MC; Romero Z; Kaufman ML; Joglekar AV; Ho M; Lumaquin D; Gray D; Lill GR; Cooper AR; Urbinati F; Senadheera S; Zhu A; Liu PQ; Paschon DE; Zhang L; Rebar EJ; Wilber A; Wang X; Gregory PD; Holmes MC; Reik A; Hollis RP; Kohn DB
    Blood; 2015 Apr; 125(17):2597-604. PubMed ID: 25733580
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Comparison of Zinc Finger Nucleases Versus CRISPR-Specific Nucleases for Genome Editing of the Wiskott-Aldrich Syndrome Locus.
    Gutierrez-Guerrero A; Sanchez-Hernandez S; Galvani G; Pinedo-Gomez J; Martin-Guerra R; Sanchez-Gilabert A; Aguilar-González A; Cobo M; Gregory P; Holmes M; Benabdellah K; Martin F
    Hum Gene Ther; 2018 Mar; 29(3):366-380. PubMed ID: 28922955
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Preclinical evaluation for engraftment of CD34
    Uchida N; Li L; Nassehi T; Drysdale CM; Yapundich M; Gamer J; Haro-Mora JJ; Demirci S; Leonard A; Bonifacino AC; Krouse AE; Linde NS; Allen C; Peshwa MV; De Ravin SS; Donahue RE; Malech HL; Tisdale JF
    Cell Rep Med; 2021 Apr; 2(4):100247. PubMed ID: 33948577
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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]  

  • 15. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
    Urbinati F; Campo Fernandez B; Masiuk KE; Poletti V; Hollis RP; Koziol C; Kaufman ML; Brown D; Mavilio F; Kohn DB
    Hum Gene Ther; 2018 Oct; 29(10):1153-1166. PubMed ID: 30198339
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation.
    Huang X; Wang Y; Yan W; Smith C; Ye Z; Wang J; Gao Y; Mendelsohn L; Cheng L
    Stem Cells; 2015 May; 33(5):1470-9. PubMed ID: 25702619
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease.
    Germino-Watnick P; Hinds M; Le A; Chu R; Liu X; Uchida N
    Cells; 2022 Jun; 11(11):. PubMed ID: 35681538
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
    Everette KA; Newby GA; Levine RM; Mayberry K; Jang Y; Mayuranathan T; Nimmagadda N; Dempsey E; Li Y; Bhoopalan SV; Liu X; Davis JR; Nelson AT; Chen PJ; Sousa AA; Cheng Y; Tisdale JF; Weiss MJ; Yen JS; Liu DR
    Nat Biomed Eng; 2023 May; 7(5):616-628. PubMed ID: 37069266
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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]  

  • 20. 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]  

    [Next]    [New Search]
    of 30.