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- [1] SPK-8011: Preliminary results from a phase 1/2 trial of investigational gene therapy for hemophilia AHAEMOPHILIA, 2018, 24 : 30 - 31George, L. A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USARagni, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Med, Med Ctr, Pittsburgh, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USASullivan, S. K.论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Pediat Hematol Oncol, Madison, MS USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USABarrett, J. C.论文数: 0 引用数: 0 h-index: 0机构: Virginia Commonwealth Univ, Cent Virginia Ctr Coagulat Disorders, Richmond, VA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USASamelson-Jones, B. J.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USACuker, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Hematol, Philadelphia, PA 19104 USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USACole, G.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAWright, F.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAChen, Y.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, 3737 Market St,Suite 1300, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAHui, D. J.论文数: 0 引用数: 0 h-index: 0机构: 3737 Market St,Suite 1300, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USALiu, Y.论文数: 0 引用数: 0 h-index: 0机构: 3737 Market St,Suite 1300, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAWachtel, K.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Regulatory Affairs, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USATakefman, D.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Regulatory Affairs, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USACouto, L. B.论文数: 0 引用数: 0 h-index: 0机构: 3737 Market St,Suite 1300, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAReape, K.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USACarr, M. E.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAAnguela, X. M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USAHigh, K. A.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Res & Dev, Philadelphia, PA USA Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA 19104 USA
- [2] Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People with Hemophilia ABLOOD, 2022, 140 : 10662 - 10664Huyen Tran论文数: 0 引用数: 0 h-index: 0机构: Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, Australia Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaEyster, M. Elaine论文数: 0 引用数: 0 h-index: 0机构: Penn State Hlth Milton S Hershey Med Ctr, Hershey, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaCroteau, Stacy E.论文数: 0 引用数: 0 h-index: 0机构: Boston Childrens Hosp, Boston Hemophilia Ctr, Boston, MA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaRagni, Margaret, V论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaSamelson-Jones, Benjamin J.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaSullivan, Spencer论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Madison, MS USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaRasko, John E. J.论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Gene & Stem Cell Therapy Program, Centenary Inst, Camperdown, NSW, Australia Univ Sydney, Fac Med & Hlth, Sydney, NSW, Australia Royal Prince Alfred Hosp, Cell & Mol Therapies, Sydney, NSW, Australia Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaJaworski, Kristen论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaMacDougall, Amy论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaJaeger, Savina论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaHofer, Magdalena论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaLi, Charlie论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaChang, Tiffany论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, AustraliaLevy, Gallia论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut Inc, Philadelphia, PA USA Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, Australia
- [3] A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of BleedsBLOOD, 2018, 132High, Katherine A.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAGeorge, Lindsey A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USAEyster, M. Elaine论文数: 0 引用数: 0 h-index: 0机构: Penn State Hlth Milton S Hershey Med Ctr, Div Hematol Oncol, Dept Med, Hershey, PA USA Spark Therapeut, Philadelphia, PA USASullivan, Spencer K.论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Jackson, MS USA Spark Therapeut, Philadelphia, PA USARagni, Margaret V.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Med & Clin Translat Sci, Pittsburgh, PA USA Spark Therapeut, Philadelphia, PA USACroteau, Stacy E.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Boston, Boston, MA USA Spark Therapeut, Philadelphia, PA USASamelson-Jones, Ben J.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USAEvans, Matthew论文数: 0 引用数: 0 h-index: 0机构: Penn State Univ, Milton S Hershey Med Ctr, Hershey, PA 17033 USA Spark Therapeut, Philadelphia, PA USAJoseney-Antoine, Marcelyne论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAMacdougall, Amy论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAKadosh, Judith论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA Hemophilia Ctr Western PA, Pittsburgh, PA USA Spark Therapeut, Philadelphia, PA USARunoski, Alexa R.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USACampbell-Baird, Cynthia论文数: 0 引用数: 0 h-index: 0机构: Penn State Univ, Milton S Hershey Med Ctr, Hershey, PA 17033 USA Spark Therapeut, Philadelphia, PA USADouglas, Kayla论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Dept Pediat, Madison, MS USA Spark Therapeut, Philadelphia, PA USATompkins, Summer论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAHait, Howard论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USACouto, Linda B.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USABassiri, Ashlyn Eaton论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAValentino, Leonard A.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USACarr, Marcus E.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAHui, Daniel J.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAWachtel, Katie论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USATakefman, Daniel论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAAnguela, Xavier M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USAReape, Kathleen B.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Spark Therapeut, Philadelphia, PA USA
- [4] SPK-8011: Preliminary Results from a Phase 1/2 Trial of Investigational Gene Therapy for Hemophilia Confirm Transgene Derived Increases in FVIII Activity That Are Persistent and Stable beyond Eight MonthsMOLECULAR THERAPY, 2018, 26 (05) : 163 - 163Sullivan, Spencer K.论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Jackson, MS USA Mississippi Ctr Adv Med, Jackson, MS USAGeorge, Lindsey A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Mississippi Ctr Adv Med, Jackson, MS USARagni, Margaret V.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA Mississippi Ctr Adv Med, Jackson, MS USASamelson-Jones, Ben论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Mississippi Ctr Adv Med, Jackson, MS USACuker, Adam论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Mississippi Ctr Adv Med, Jackson, MS USAChen, Yifeng论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USAHui, Daniel J.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USAWachtel, Katie论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USATakefman, Daniel论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USACouto, Linda B.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USACarr, Marcus E.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USAReape, Kathleen Z.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USAAnguela, Xavier M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USAHigh, Katherine A.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Jackson, MS USA
- [5] SPK-8016: PRELIMINARY RESULTS FROM A PHASE 1/2 CLINICAL TRIAL OF GENE THERAPY FOR HEMOPHILIA AHAEMOPHILIA, 2021, 27 : 129 - 130Sullivan, S. K.论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Madison, MS USA Mississippi Ctr Adv Med, Madison, MS USABarrett, J. C.论文数: 0 引用数: 0 h-index: 0机构: Virginia Commonwealth Univ, Dept Internal Med, Div Hematol Oncol, Richmond, VA USA Mississippi Ctr Adv Med, Madison, MS USADrelich, D. A.论文数: 0 引用数: 0 h-index: 0机构: Thomas Jefferson Univ, Dept Med, Cardeza Fdn Hematol Res, Philadelphia, PA 19107 USA Mississippi Ctr Adv Med, Madison, MS USATarantino, M. D.论文数: 0 引用数: 0 h-index: 0机构: Univ Illinois, Coll Med, Peoria, IL 61656 USA Bleeding & Clotting Disorders Inst, Peoria, IL USA Mississippi Ctr Adv Med, Madison, MS USAMacDougall, A.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAJoseney-Antoine, M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAWachtel, K.论文数: 0 引用数: 0 h-index: 0机构: Akouos, Boston, MA USA Mississippi Ctr Adv Med, Madison, MS USAJaworski, K.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USACurran, M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAKuranda, K.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAArmour, S. M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAMingozzi, F.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAAnguela, X. M.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAReape, K. Z.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAMonahan, P. E.论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Mississippi Ctr Adv Med, Madison, MS USAHigh, K. A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pediat, Perelman Sch Med, Philadelphia, PA 19104 USA Mississippi Ctr Adv Med, Madison, MS USA
- [6] Long-Term Durable FVIII Expression with Improvements in Bleeding Rates Following AAV-Mediated FVIII Gene Transfer for Hemophilia A: Multiyear Follow-up on the Phase I/II Trial of SPK-8011BLOOD, 2022, 140 : 1899 - 1901Croteau, Stacy E.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Dept Pediat, Boston, MA USA Boston Childrens Hosp, Div Hematol & Oncol, Boston, MA USA Harvard Med Sch, Dept Pediat, Boston, MA USAEyster, M. Elaine论文数: 0 引用数: 0 h-index: 0机构: Penn State Hlth Milton S Hershey Med Ctr, Dept Med, Div Hematol & Oncol, Hershey, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USAHuyen Tran论文数: 0 引用数: 0 h-index: 0机构: Monash Univ, Australian Ctr Blood Dis, Melbourne, Vic, Australia Harvard Med Sch, Dept Pediat, Boston, MA USARagni, Margaret, V论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Med, Pittsburgh, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USASamelson-Jones, Benjamin J.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Div Hematol, Philadelphia, PA USA Childrens Hosp, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Harvard Med Sch, Dept Pediat, Boston, MA USAGeorge, Lindsey论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Div Hematol, Philadelphia, PA USA Childrens Hosp, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Harvard Med Sch, Dept Pediat, Boston, MA USASullivan, Spencer论文数: 0 引用数: 0 h-index: 0机构: Mississippi Ctr Adv Med, Madison, MS USA Harvard Med Sch, Dept Pediat, Boston, MA USARasko, John E. J.论文数: 0 引用数: 0 h-index: 0机构: Royal Prince Alfred Hosp, Dept Cell & Mol Therapies, Sydney, NSW, Australia Univ Sydney, Fac Med & Hlth, Centenary Inst, Gene & Stem Cell Therapy Program, Sydney, NSW, Australia Harvard Med Sch, Dept Pediat, Boston, MA USAMoormeier, Jill论文数: 0 引用数: 0 h-index: 0机构: UMKC Sch Med, Partment Med, Kansas City, MO USA Harvard Med Sch, Dept Pediat, Boston, MA USAAngchaisuksiri, Pantep论文数: 0 引用数: 0 h-index: 0机构: Mahidol Univ, Ramathibodi Hosp, Dept Med, Bangkok, Thailand Univ N Carolina, Sch Med, Chapel Hill, NC USA Harvard Med Sch, Dept Pediat, Boston, MA USATeitel, Jerome论文数: 0 引用数: 0 h-index: 0机构: Univ Toronto, St Michaels Hosp Haemophilia Treatment Ctr, Toronto, ON, Canada Harvard Med Sch, Dept Pediat, Boston, MA USAKenet, Gili论文数: 0 引用数: 0 h-index: 0机构: Sheba Med Ctr, Israel Natl Hemophilia Ctr, Tel Hashomer, Israel Sheba Med Ctr, Thrombosis Inst, Tel Hashomer, Israel Harvard Med Sch, Dept Pediat, Boston, MA USAWynn, Tung论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Hematol & Oncol, Gainesville, FL USA Harvard Med Sch, Dept Pediat, Boston, MA USAJaworski, Kristen论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USAMacdougall, Amy论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USAJaeger, Savina论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USATrivedi, Trupti论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USAMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USAChang, Tiffany论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USALevy, Gallia论文数: 0 引用数: 0 h-index: 0机构: Spark Therapeut, Philadelphia, PA USA Harvard Med Sch, Dept Pediat, Boston, MA USA
- [7] The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapyBIOTECHNOLOGY LETTERS, 2009, 31 (03) : CP7 - CP14Shen Youjin论文数: 0 引用数: 0 h-index: 0机构: Shantou Univ, Coll Med, Hosp 2, Dept Hematol, Shantou 515041, Peoples R China Shantou Univ, Coll Med, Hosp 2, Dept Hematol, Shantou 515041, Peoples R ChinaYin Jun论文数: 0 引用数: 0 h-index: 0机构: Shantou Univ, Coll Med, Hosp 2, Dept Lab, Shantou 515041, Peoples R China Shantou Univ, Coll Med, Hosp 2, Dept Hematol, Shantou 515041, Peoples R China
- [8] The treatment of hemophilia A: from protein replacement to AAV-mediated gene therapyBiotechnology Letters, 2009, 31 : 321 - 328Shen Youjin论文数: 0 引用数: 0 h-index: 0机构: The Second Hospital of Shantou University Medical College,Department of HematologyYin Jun论文数: 0 引用数: 0 h-index: 0机构: The Second Hospital of Shantou University Medical College,Department of Hematology
- [9] A phase I trial of AAV-mediated muscle-directed gene therapy for hemophilia B.BLOOD, 1999, 94 (10) : 642A - 642AManno, CS论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAHerzog, RW论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAArruda, VR论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USACouto, LB论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USATai, SJ论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAMcClelland, A论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAFlake, AW论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAChew, AJ论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAFields, PA论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAArmstrong, AE论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USALeonard, D论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USASkarsgard, ED论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAGlader, BE论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USARagni, MV论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USALarson, PJ论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAKay, MA论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USAHigh, KA论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Med Ctr, Philadelphia, PA 19104 USA
- [10] A phase I trial of AAV-mediated muscle directed gene transfer for hemophilia B.BLOOD, 2000, 96 (11) : 801A - 801AManno, CS论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAGlader, B论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USARagni, MV论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAThompson, A论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USACosta, FF论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAChew, AJ论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAHerzog, RW论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAArruda, VR论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USACouto, LB论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAClelland, AM论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAJohnson, F论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAFlake, A论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USASkarsgard, E论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAArmstrong, E论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAKay, M论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAHigh, KA论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA