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- [1] Human liver biopsy analysis shows decline in FVIII levels following AAV5-hFVIII-SQ gene therapy may be due to low RNA transcription levels despite persistence of full-length episomal vector genomesHAEMOPHILIA, 2024, 30 : 56 - 58Yates, Bridget论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAIsmail, Ashrafali论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAMattis, Aras论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Francisco, Dept Med, Ctr Liver, San Francisco, CA USA Univ Calif San Francisco, Dept Pathol, San Francisco, CA USA BioMarin Pharmaceut Inc, Novato, CA USAGonzalez, Alyssa论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAKim, Chan Kyu论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAJayaram, Kala论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAKenet, Gili论文数: 0 引用数: 0 h-index: 0机构: Sackler Sch Med, Amalia Biron Res Inst Thrombosis & Hemostasis, Tel Aviv, Israel BioMarin Pharmaceut Inc, Novato, CA USAMason, Jane论文数: 0 引用数: 0 h-index: 0机构: Univ Queensland, Royal Brisbane & Womens Hosp, Queensland Haemophilia Ctr, Canc Care Serv, Brisbane, Qld, Australia BioMarin Pharmaceut Inc, Novato, CA USAMahlangu, Johnny论文数: 0 引用数: 0 h-index: 0机构: Charlotte Maxeke Johannesburg Acad Hosp, Johannesburg, South Africa BioMarin Pharmaceut Inc, Novato, CA USADunn, Amy论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Div Hematol Oncol & Blood & Marrow Transplant, Columbus, OH USA Ohio State Univ, Coll Med, Columbus, OH USA BioMarin Pharmaceut Inc, Novato, CA USAShapiro, Susan论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Oxford Haemophilia & Thrombosis Ctr, NIHR Oxford Biomed Res Ctr, Oxford Univ Hosp NHS Fdn Trust Radcliffe,Dept Med, Oxford, England BioMarin Pharmaceut Inc, Novato, CA USAWang, Michael论文数: 0 引用数: 0 h-index: 0机构: Univ Witwatersrand, Natl Hlth Lab Serv, Johannesburg, South Africa Univ Colorado, Hemophilia & Thrombosis Ctr, Sch Med, Aurora, CO USA BioMarin Pharmaceut Inc, Novato, CA USA论文数: 引用数: h-index:机构:Giermasz, Adam论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Hemophilia Treatment Ctr, Sacramento, CA USA BioMarin Pharmaceut Inc, Novato, CA USAKazmi, Rashid论文数: 0 引用数: 0 h-index: 0机构: London Southampton Univ Hosp, Southampton, Hants, England Natl Inst Hlth & Care Res Clin Res Facil, Southampton, Hants, England BioMarin Pharmaceut Inc, Novato, CA USAKey, Nigel S.论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, UNC Blood Res Ctr, Chapel Hill, NC USA BioMarin Pharmaceut Inc, Novato, CA USAOzelo, Margareth论文数: 0 引用数: 0 h-index: 0机构: Univ Estadual Campinas, UNICAMP, Hemoctr, Campinas, Brazil BioMarin Pharmaceut Inc, Novato, CA USARobinson, Tara M.论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USAFong, Sylvia论文数: 0 引用数: 0 h-index: 0机构: BioMarin Pharmaceut Inc, Novato, CA USA BioMarin Pharmaceut Inc, Novato, CA USA