BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

171 related articles for article (PubMed ID: 29719266)

  • 1. Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.
    Ramaswamy S; Tonnu N; Menon T; Lewis BM; Green KT; Wampler D; Monahan PE; Verma IM
    Cell Rep; 2018 May; 23(5):1565-1580. PubMed ID: 29719266
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system.
    Lyu C; Shen J; Wang R; Gu H; Zhang J; Xue F; Liu X; Liu W; Fu R; Zhang L; Li H; Zhang X; Cheng T; Yang R; Zhang L
    Stem Cell Res Ther; 2018 Apr; 9(1):92. PubMed ID: 29625575
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Hepatocyte-Like Cells Derived From Mouse Induced Pluripotent Stem Cells Produce Functional Coagulation Factor IX in a Hemophilia B Mouse Model.
    Wu YM; Huang YJ; Chen P; Hsu YC; Lin SW; Lai HS; Lee HS
    Cell Transplant; 2016; 25(7):1237-46. PubMed ID: 26395869
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Genetic Correction and Hepatic Differentiation of Hemophilia B-specific Human Induced Pluripotent Stem Cells.
    He Q; Wang HH; Cheng T; Yuan WP; Ma YP; Jiang YP; Ren ZH
    Chin Med Sci J; 2017 Sep; 32(3):135-144. PubMed ID: 28956740
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Genetic modification of donor hepatocytes improves therapeutic efficacy for hemophilia B in mice.
    Wu YM; Kao CY; Huang YJ; Yu IS; Lee HS; Lai HS; Lee PH; Lin CN; Lin SW
    Cell Transplant; 2010; 19(9):1169-80. PubMed ID: 20412633
    [TBL] [Abstract][Full Text] [Related]  

  • 6. In vitro recovery of FIX clotting activity as a marker of highly functional hepatocytes in a hemophilia B iPSC model.
    Luce E; Steichen C; Allouche M; Messina A; Heslan JM; Lambert T; Weber A; Nguyen TH; Christophe O; Dubart-Kupperschmitt A
    Hepatology; 2022 Apr; 75(4):866-880. PubMed ID: 34687060
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Therapeutic effects of hepatocyte transplantation on hemophilia B.
    Tatsumi K; Ohashi K; Shima M; Nakajima Y; Okano T; Yoshioka A
    Transplantation; 2008 Jul; 86(1):167-70. PubMed ID: 18622295
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Advanced cell-based modeling of the royal disease: characterization of the mutated F9 mRNA.
    Martorell L; Luce E; Vazquez JL; Richaud-Patin Y; Jimenez-Delgado S; Corrales I; Borras N; Casacuberta-Serra S; Weber A; Parra R; Altisent C; Follenzi A; Dubart-Kupperschmitt A; Raya A; Vidal F; Barquinero J
    J Thromb Haemost; 2017 Nov; 15(11):2188-2197. PubMed ID: 28834196
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Successful in vivo propagation of factor IX-producing hepatocytes in mice: potential for cell-based therapy in haemophilia B.
    Tatsumi K; Ohashi K; Kataoka M; Tateno C; Shibata M; Naka H; Shima M; Hisanaga M; Kanehiro H; Okano T; Yoshizato K; Nakajima Y; Yoshioka A
    Thromb Haemost; 2008 May; 99(5):883-91. PubMed ID: 18449417
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Human iPS Cell-based Liver-like Tissue Engineering at Extrahepatic Sites in Mice as a New Cell Therapy for Hemophilia B.
    Okamoto R; Takayama K; Akita N; Nagamoto Y; Hosokawa D; Iizuka S; Sakurai F; Suemizu H; Ohashi K; Mizuguchi H
    Cell Transplant; 2018 Feb; 27(2):299-309. PubMed ID: 29637813
    [TBL] [Abstract][Full Text] [Related]  

  • 11. CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs.
    Morishige S; Mizuno S; Ozawa H; Nakamura T; Mazahery A; Nomura K; Seki R; Mouri F; Osaki K; Yamamura K; Okamura T; Nagafuji K
    Int J Hematol; 2020 Feb; 111(2):225-233. PubMed ID: 31664646
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Propagating factor IX-producing hepatocytes for haemophilia B therapy.
    Ofosu FA
    Thromb Haemost; 2008 May; 99(5):799-800. PubMed ID: 18449406
    [No Abstract]   [Full Text] [Related]  

  • 13. Systemic delivery of factor IX messenger RNA for protein replacement therapy.
    Ramaswamy S; Tonnu N; Tachikawa K; Limphong P; Vega JB; Karmali PP; Chivukula P; Verma IM
    Proc Natl Acad Sci U S A; 2017 Mar; 114(10):E1941-E1950. PubMed ID: 28202722
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Creation of a mouse expressing defective human factor IX.
    Jin DY; Zhang TP; Gui T; Stafford DW; Monahan PE
    Blood; 2004 Sep; 104(6):1733-9. PubMed ID: 15178576
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.
    Monahan PE; Sun J; Gui T; Hu G; Hannah WB; Wichlan DG; Wu Z; Grieger JC; Li C; Suwanmanee T; Stafford DW; Booth CJ; Samulski JJ; Kafri T; McPhee SW; Samulski RJ
    Hum Gene Ther; 2015 Feb; 26(2):69-81. PubMed ID: 25419787
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Paired CRISPR/Cas9 Nickases Mediate Efficient Site-Specific Integration of
    Wang Y; Zhao J; Duan N; Liu W; Zhang Y; Zhou M; Hu Z; Feng M; Liu X; Wu L; Li Z; Liang D
    Int J Mol Sci; 2018 Oct; 19(10):. PubMed ID: 30301136
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Modeling of hemophilia A using patient-specific induced pluripotent stem cells derived from urine cells.
    Jia B; Chen S; Zhao Z; Liu P; Cai J; Qin D; Du J; Wu C; Chen Q; Cai X; Zhang H; Yu Y; Pei D; Zhong M; Pan G
    Life Sci; 2014 Jul; 108(1):22-9. PubMed ID: 24834837
    [TBL] [Abstract][Full Text] [Related]  

  • 18. CRISPR-Mediated In Situ Introduction or Integration of
    Tang Q; Hu Z; Zhao J; Zhou T; Tang S; Wang P; Xiao R; Chen Y; Wu L; Zhou M; Liang D
    Int J Mol Sci; 2023 May; 24(10):. PubMed ID: 37240366
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Clinical progress in gene therapy: sustained partial correction of the bleeding disorder in patients suffering from severe hemophilia B.
    Vandendriessche T; Chuah MK
    Hum Gene Ther; 2012 Jan; 23(1):4-6. PubMed ID: 22149214
    [No Abstract]   [Full Text] [Related]  

  • 20. Hepatic control elements promote long-term expression of human coagulation factor IX gene in hydrodynamically transfected mice.
    Kim HS; Kim JC; Lee YK; Kim JS; Park YS
    J Gene Med; 2011 Jul; 13(7-8):365-72. PubMed ID: 21710610
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.