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 *

711 related articles for article (PubMed ID: 31893514)

  • 1. Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
    Pasi KJ; Rangarajan S; Mitchell N; Lester W; Symington E; Madan B; Laffan M; Russell CB; Li M; Pierce GF; Wong WY
    N Engl J Med; 2020 Jan; 382(1):29-40. PubMed ID: 31893514
    [TBL] [Abstract][Full Text] [Related]  

  • 2. AAV5-Factor VIII Gene Transfer in Severe Hemophilia A.
    Rangarajan S; Walsh L; Lester W; Perry D; Madan B; Laffan M; Yu H; Vettermann C; Pierce GF; Wong WY; Pasi KJ
    N Engl J Med; 2017 Dec; 377(26):2519-2530. PubMed ID: 29224506
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
    Ozelo MC; Mahlangu J; Pasi KJ; Giermasz A; Leavitt AD; Laffan M; Symington E; Quon DV; Wang JD; Peerlinck K; Pipe SW; Madan B; Key NS; Pierce GF; O'Mahony B; Kaczmarek R; Henshaw J; Lawal A; Jayaram K; Huang M; Yang X; Wong WY; Kim B;
    N Engl J Med; 2022 Mar; 386(11):1013-1025. PubMed ID: 35294811
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
    George LA; Monahan PE; Eyster ME; Sullivan SK; Ragni MV; Croteau SE; Rasko JEJ; Recht M; Samelson-Jones BJ; MacDougall A; Jaworski K; Noble R; Curran M; Kuranda K; Mingozzi F; Chang T; Reape KZ; Anguela XM; High KA
    N Engl J Med; 2021 Nov; 385(21):1961-1973. PubMed ID: 34788507
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.
    Fong S; Yates B; Sihn CR; Mattis AN; Mitchell N; Liu S; Russell CB; Kim B; Lawal A; Rangarajan S; Lester W; Bunting S; Pierce GF; Pasi KJ; Wong WY
    Nat Med; 2022 Apr; 28(4):789-797. PubMed ID: 35411075
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
    Mahlangu J; Kaczmarek R; von Drygalski A; Shapiro S; Chou SC; Ozelo MC; Kenet G; Peyvandi F; Wang M; Madan B; Key NS; Laffan M; Dunn AL; Mason J; Quon DV; Symington E; Leavitt AD; Oldenburg J; Chambost H; Reding MT; Jayaram K; Yu H; Mahajan R; Chavele KM; Reddy DB; Henshaw J; Robinson TM; Wong WY; Pipe SW;
    N Engl J Med; 2023 Feb; 388(8):694-705. PubMed ID: 36812433
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A.
    Long BR; Veron P; Kuranda K; Hardet R; Mitchell N; Hayes GM; Wong WY; Lau K; Li M; Hock MB; Zoog SJ; Vettermann C; Mingozzi F; Schweighardt B
    Mol Ther; 2021 Feb; 29(2):597-610. PubMed ID: 33309883
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy.
    Fonck C; Su C; Arens J; Koziol E; Srimani J; Henshaw J; Van Tuyl A; Chandra S; Vettermann C; O'Neill CA
    Gene Ther; 2023 Aug; 30(7-8):581-586. PubMed ID: 35132205
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
    George LA; Sullivan SK; Giermasz A; Rasko JEJ; Samelson-Jones BJ; Ducore J; Cuker A; Sullivan LM; Majumdar S; Teitel J; McGuinn CE; Ragni MV; Luk AY; Hui D; Wright JF; Chen Y; Liu Y; Wachtel K; Winters A; Tiefenbacher S; Arruda VR; van der Loo JCM; Zelenaia O; Takefman D; Carr ME; Couto LB; Anguela XM; High KA
    N Engl J Med; 2017 Dec; 377(23):2215-2227. PubMed ID: 29211678
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice.
    Greig JA; Wang Q; Reicherter AL; Chen SJ; Hanlon AL; Tipper CH; Clark KR; Wadsworth S; Wang L; Wilson JM
    Hum Gene Ther; 2017 May; 28(5):392-402. PubMed ID: 28056565
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec.
    Zhang L; Handyside B; Murphy R; Sihn CR; Xie L; Vitelli C; Harmon D; Sisó S; Liu S; Bullens S; Bunting S; Fong S
    Mol Ther Methods Clin Dev; 2020 Jun; 17():13-20. PubMed ID: 31890737
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A.
    Malec L; Peyvandi F; Chan AKC; Königs C; Zulfikar B; Yuan H; Simpson M; Álvarez Román MT; Carcao M; Staber JM; Dunn AL; Chou SC; d'Oiron R; Albisetti M; Demissie M; Santagostino E; Yarramaneni A; Wong N; Abad-Franch L; Gunawardena S; Fijnvandraat K;
    N Engl J Med; 2024 Jul; 391(3):235-246. PubMed ID: 39018533
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
    von Drygalski A; Chowdary P; Kulkarni R; Susen S; Konkle BA; Oldenburg J; Matino D; Klamroth R; Weyand AC; Jimenez-Yuste V; Nogami K; Poloskey S; Winding B; Willemze A; Knobe K;
    N Engl J Med; 2023 Jan; 388(4):310-318. PubMed ID: 36720133
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
    Nathwani AC; Reiss UM; Tuddenham EG; Rosales C; Chowdary P; McIntosh J; Della Peruta M; Lheriteau E; Patel N; Raj D; Riddell A; Pie J; Rangarajan S; Bevan D; Recht M; Shen YM; Halka KG; Basner-Tschakarjan E; Mingozzi F; High KA; Allay J; Kay MA; Ng CY; Zhou J; Cancio M; Morton CL; Gray JT; Srivastava D; Nienhuis AW; Davidoff AM
    N Engl J Med; 2014 Nov; 371(21):1994-2004. PubMed ID: 25409372
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors.
    Mahlangu J; Oldenburg J; Paz-Priel I; Negrier C; Niggli M; Mancuso ME; Schmitt C; Jiménez-Yuste V; Kempton C; Dhalluin C; Callaghan MU; Bujan W; Shima M; Adamkewicz JI; Asikanius E; Levy GG; Kruse-Jarres R
    N Engl J Med; 2018 Aug; 379(9):811-822. PubMed ID: 30157389
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Long-term follow-up of liver-directed, adeno-associated vector-mediated gene therapy in the canine model of hemophilia A.
    Batty P; Mo AM; Hurlbut D; Ishida J; Yates B; Brown C; Harpell L; Hough C; Pender A; Rimmer EK; Sardo Infirri S; Winterborn A; Fong S; Lillicrap D
    Blood; 2022 Dec; 140(25):2672-2683. PubMed ID: 35405003
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A.
    Shima M; Hanabusa H; Taki M; Matsushita T; Sato T; Fukutake K; Fukazawa N; Yoneyama K; Yoshida H; Nogami K
    N Engl J Med; 2016 May; 374(21):2044-53. PubMed ID: 27223146
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy.
    Rosen S; Tiefenbacher S; Robinson M; Huang M; Srimani J; Mackenzie D; Christianson T; Pasi KJ; Rangarajan S; Symington E; Giermasz A; Pierce GF; Kim B; Zoog SJ; Vettermann C
    Blood; 2020 Nov; 136(22):2524-2534. PubMed ID: 32915950
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
    Zhang L; Yates B; Murphy R; Liu S; Xie L; Handyside B; Sihn CR; Bouwman T; Galicia N; Tan D; Fonck C; Arens J; Clark A; Zhang W; Chandra S; Srimani J; Holcomb J; Van Tuyl A; Henshaw J; Vettermann C; Siso S; Su C; Bullens S; Bunting S; O'Neill C; Fong S
    Mol Ther Methods Clin Dev; 2022 Sep; 26():519-531. PubMed ID: 36092364
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers.
    Sihn CR; Handyside B; Liu S; Zhang L; Murphy R; Yates B; Xie L; Torres R; Russell CB; O'Neill CA; Pungor E; Bunting S; Fong S
    Mol Ther Methods Clin Dev; 2022 Mar; 24():142-153. PubMed ID: 35036471
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 36.