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 *

422 related articles for article (PubMed ID: 31147717)

  • 1. 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]  

  • 2. 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]  

  • 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. Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing.
    Wen J; Tao W; Hao S; Zu Y
    J Hematol Oncol; 2017 Jun; 10(1):119. PubMed ID: 28610635
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. 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]  

  • 7. 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]  

  • 8. 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]  

  • 9. 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]  

  • 10. 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]  

  • 11. 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]  

  • 12. 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]  

  • 13. 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]  

  • 14. 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]  

  • 15. 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]  

  • 16. 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]  

  • 17. 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]  

  • 18. 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]  

  • 19. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
    Ramadier S; Chalumeau A; Felix T; Othman N; Aknoun S; Casini A; Maule G; Masson C; De Cian A; Frati G; Brusson M; Concordet JP; Cavazzana M; Cereseto A; El Nemer W; Amendola M; Wattellier B; Meneghini V; Miccio A
    Mol Ther; 2022 Jan; 30(1):145-163. PubMed ID: 34418541
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 22.