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- [21] Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal NeovascularizationMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 409 - 417Chung, Sook Hyun论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAMollhoff, Iris Natalie论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USANguyen, Uyen论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USANguyen, Amy论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAStucka, Natalie论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USATieu, Eric论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAManna, Suman论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAMeleppat, Ratheesh Kumar论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAZhang, Pengfei论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USANguyen, Emerald Lovece论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAFong, Jared论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAZawadzki, Robert论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USAYiu, Glenn论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA Univ Calif Davis, Dept Ophthalmol & Vis Sci, Davis, CA 95616 USA
- [22] Single AAV-mediated mutation replacement genome editing in limited number of photoreceptors restores vision in miceNature Communications, 11Koji M. Nishiguchi论文数: 0 引用数: 0 h-index: 0机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic MedicineKosuke Fujita论文数: 0 引用数: 0 h-index: 0机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic MedicineFuyuki Miya论文数: 0 引用数: 0 h-index: 0机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic MedicineShota Katayama论文数: 0 引用数: 0 h-index: 0机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic MedicineToru Nakazawa论文数: 0 引用数: 0 h-index: 0机构: Tohoku University Graduate School of Medicine,Department of Advanced Ophthalmic Medicine
- [23] Emerging Issues in AAV-Mediated In Vivo Gene TherapyMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2018, 8 : 87 - 104Colella, Pasqualina论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, FranceRonzitti, Giuseppe论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, FranceMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France Univ Pierre & Marie Curie Paris 6, F-75651 Paris, France INSERM U974, F-75651 Paris, France Univ Evry, Univ Paris Saclay, Genethon, INSERM INTEGRARE U951, F-91001 Evry, France
- [24] Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemiaPLOS ONE, 2022, 17 (09):Zhang, Shengwen论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USABastille, Amy论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAGordo, Susana论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USARamesh, Nikhil论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAVora, Jenisha论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAMcCarthy, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAZhang, Xiaohan论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAFrank, Dylan论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAKo, Chih-Wei论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAWu, Carmen论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAWalsh, Noel论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAAmarwani, Shreya论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USALiao, Jing论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAXiong, Qiang论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USADrouin, Lauren论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAHebben, Matthias论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAChiang, Kyle论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USAChau, B. Nelson论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut Inc, Lexington, MA 02421 USA LogicBio Therapeut Inc, Lexington, MA 02421 USA
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- [27] Standard screening methods underreport AAV-mediated transduction and gene editingNATURE COMMUNICATIONS, 2019, 10 (1)Lang, Jonathan F.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, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USAToulmin, Sushila 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, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USABrida, Kasey L.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 USAEisenlohr, Laurence C.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, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USADavidson, Beverly L.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, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
- [28] Nuclease-free, promoterless recombinant AAV-mediated genome editing restores function of hepatocytes leading to selective advantage and repopulation in mouse models with liver diseaseHUMAN GENE THERAPY, 2021, 32 (19-20) : A16 - A16Zhang, S.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USABastille, A.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USARamesh, N.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAMcCarthy, E.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAVora, J.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAKo, C.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAWu, C.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAWalsh, N.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAFrank, D.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAWang, C. C.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAWang, S.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAWang, J.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAFreland, L.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USALiao, J.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAGordo, S.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAXiong, Q.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USADrouin, L.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAPreston, G.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAHebben, M.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAChiang, K.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USAChau, N.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USANacht, M.论文数: 0 引用数: 0 h-index: 0机构: LogicBio Therapeut, Lexington, MA USA LogicBio Therapeut, Lexington, MA USA
- [29] Standard screening methods underreport AAV-mediated transduction and gene editingNature Communications, 10Jonathan F. Lang论文数: 0 引用数: 0 h-index: 0机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of MedicineSushila A. Toulmin论文数: 0 引用数: 0 h-index: 0机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of MedicineKasey L. Brida论文数: 0 引用数: 0 h-index: 0机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of MedicineLaurence C. Eisenlohr论文数: 0 引用数: 0 h-index: 0机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of MedicineBeverly L. Davidson论文数: 0 引用数: 0 h-index: 0机构: The Children’s Hospital of Philadelphia,The Department of Pathology and Laboratory Medicine, Perelman School of Medicine
- [30] Impact of liver fibrosis on AAV-mediated gene transfer to mouse hepatocytesNATURE COMMUNICATIONS, 2025, 16 (01)Ferriero, Rosa论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyBruno, Gemma论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPadula, Agnese论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPisano, Simone论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy European Sch Mol Med SEMM, Milan, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyBoffa, Iolanda论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyGargaro, Marco论文数: 0 引用数: 0 h-index: 0机构: Univ Perugia, Dept Med & Surg, Perugia, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyImperatore, Teresa论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Sch Adv Studies, Scuola Super Meridionale, Genom & Expt Med Program, SSM, Naples, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyBattipaglia, Maria论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy European Sch Mol Med SEMM, Milan, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyVivenzio, Silvia论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPerna, Claudia论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyNusco, Edoardo论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyFerrante, Luigi论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyWesthaus, Adrian论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Childrens Med Res Inst, Fac Med & Hlth, Translat Vectorol Res Unit, Westmead, NSW, Australia INSERM, Integrare Res Unit, UMR S951, Evry, France Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyKnight, Maddison论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Childrens Med Res Inst, Fac Med & Hlth, Translat Vectorol Res Unit, Westmead, NSW, Australia Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyManni, Giorgia论文数: 0 引用数: 0 h-index: 0机构: Univ Perugia, Dept Med & Surg, Perugia, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyCampione, Severo论文数: 0 引用数: 0 h-index: 0机构: Cardarelli Hosp, Pathol Unit, Naples, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyDi Napoli, Evaristo论文数: 0 引用数: 0 h-index: 0机构: Feder II Univ Naples, Dept Vet Med & Anim Prod, Naples, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPolishchuk, Elena论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPolishchuk, Roman论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyPaciello, Orlando论文数: 0 引用数: 0 h-index: 0机构: Feder II Univ Naples, Dept Vet Med & Anim Prod, Naples, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyBrunetti-Pierri, Nicola论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Sch Adv Studies, Scuola Super Meridionale, Genom & Expt Med Program, SSM, Naples, Italy Federico II Univ Naples, Dept Translat Med, Naples, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, ItalyLisowski, Leszek论文数: 0 引用数: 0 h-index: 0机构: Univ Sydney, Childrens Med Res Inst, Fac Med & Hlth, Translat Vectorol Res Unit, Westmead, NSW, Australia Sydney Childrens Hosp Network, Childrens Med Res Inst, Australian Genome Therapeut Ctr, Westmead, NSW, Australia Mil Inst Med Natl Res Inst, Lab Mol Oncol & Innovat Therapies, Warsaw, Poland Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy论文数: 引用数: h-index:机构:Piccolo, Pasquale论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy Telethon Inst Genet & Med TIGEM, Pozzuoli, Italy