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- [21] Optimization of the Capsid of Recombinant Adeno-Associated Virus 6 (AAV6) Vectors for Liver-, and Muscle-Directed Gene TherapyMOLECULAR THERAPY, 2015, 23 : S127 - S127Nolasco, Diego论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USAVan Vliet, Kim论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Biochem & Mol Biol, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USAMarkusic, David论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USALing, Chen论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USAHuang, Lin-Ya论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Biochem & Mol Biol, Gainesville, FL 32610 USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USAAgbandje-McKenna, Mavis论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Biochem & Mol Biol, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USASrivastava, Arun论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USAAslanidi, George论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL USA Univ Florida, Dept Pediat, Div Cellular & Mol Therapy, Gainesville, FL USA
- [22] 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
- [23] Systemic protein delivery by muscle-directed gene transfer is limited by a local immune responseMOLECULAR THERAPY, 2004, 9 : S71 - S71Wang, LX论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USADobrzynski, E论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USACao, O论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAHerzog, R论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
- [24] Identifying Muscle Stem Cell Tropic AAV Variants through Directed EvolutionMOLECULAR THERAPY, 2024, 32 (04) : 265 - 265Chen, Harry M.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAEmami, Michael R.论文数: 0 引用数: 0 h-index: 0机构: MyoGene Bio, San Diego, CA USA Univ Calif Los Angeles, Los Angeles, CA USAYoung, Courtney论文数: 0 引用数: 0 h-index: 0机构: MyoGene Bio, San Diego, CA USA Univ Calif Los Angeles, Los Angeles, CA USALiu, Kevin论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAFreitas, Braeden论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USASkuratovsky, Emily论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USARando, Thomas A.论文数: 0 引用数: 0 h-index: 0机构: Stanford, Palo Alto, CA USA Univ Calif Los Angeles, Los Angeles, CA USAIkotun, Oluwatayo论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAPyle, April D.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USASpencer, Melissa J.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USA
- [25] Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse modelMOLECULAR GENETICS AND METABOLISM, 2021, 134 (1-2) : 139 - 146Greig, Jenny A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USAJennis, Matthew论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USADandekar, Aditya论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USAChorazeczewski, Joanna K.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USASmith, Melanie K.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USAAshley, Scott N.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USAYan, Hanying论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USAWilson, James M.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Gene Therapy Program, Philadelphia, PA 19104 USA
- [26] Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapyMOLECULAR METABOLISM, 2024, 81Munoz, Sergio论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainBertolin, Joan论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainJimenez, Veronica论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainJaen, Maria Luisa论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainGarcia, Miquel论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainPujol, Anna论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainVila, Laia论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainSacristan, Victor论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainBarbon, Elena论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, INTEGRARE, Genethon,INSERM,UMR951, F-91002 Evry, France Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainRonzitti, Giuseppe论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, INTEGRARE, Genethon,INSERM,UMR951, F-91002 Evry, France Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainEl Andari, Jihad论文数: 0 引用数: 0 h-index: 0机构: Heidelberg Univ, Med Fac, BioQuant Ctr, Dept Infect Dis Virol,Sect Viral Vector Technol, D-69120 Heidelberg, Germany Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainTulalamba, Warut论文数: 0 引用数: 0 h-index: 0机构: Vrije Univ Brussel VUB, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium Univ Leuven, Ctr Mol & Vasc Biol, Dept Cardiovasc Sci, B-3000 Leuven, Belgium Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainPham, Quang Hong论文数: 0 引用数: 0 h-index: 0机构: Vrije Univ Brussel VUB, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium Univ Leuven, Ctr Mol & Vasc Biol, Dept Cardiovasc Sci, B-3000 Leuven, Belgium Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainRuberte, Jesus论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Sch Vet Med, Dept Anim Hlth & Anat, Bellaterra 08193, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainVandenDriessche, Thierry论文数: 0 引用数: 0 h-index: 0机构: Vrije Univ Brussel VUB, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium Univ Leuven, Ctr Mol & Vasc Biol, Dept Cardiovasc Sci, B-3000 Leuven, Belgium Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainChuah, Marinee K.论文数: 0 引用数: 0 h-index: 0机构: Vrije Univ Brussel VUB, Dept Gene Therapy & Regenerat Med, B-1090 Brussels, Belgium Univ Leuven, Ctr Mol & Vasc Biol, Dept Cardiovasc Sci, B-3000 Leuven, Belgium Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainGrimm, Dirk论文数: 0 引用数: 0 h-index: 0机构: Heidelberg Univ, Med Fac, BioQuant Ctr, Dept Infect Dis Virol,Sect Viral Vector Technol, D-69120 Heidelberg, Germany German Ctr Infect Res DZIF, Heidelberg, Germany German Ctr Cardiovasc Res DZHK, Partner Site Heidelberg, Heidelberg, Germany Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, INTEGRARE, Genethon,INSERM,UMR951, F-91002 Evry, France Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, SpainBosch, Fatima论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Dept Biochem & Mol Biol, Bellaterra 08193, Spain Inst Salud Carlos III, Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy CBATEG, Bellaterra 08193, Spain
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- [29] Differential Host T Cell Response to the Capsid of AAV2 and 8 in Muscle-Directed Gene Transfer in Murine and Non-Human Primate ModelsMOLECULAR THERAPY, 2006, 13 : S376 - S376Wang, Lili论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USACalcedo, Roberto论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USAGrant, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USAFigueredo, Joanita M.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USAWilson, James M.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA USA
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