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- [1] AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfectaMOLECULAR THERAPY NUCLEIC ACIDS, 2024, 35 (01):Yang, Yeon-Suk论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USASato, Tadatoshi论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USA UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01655 USA UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, Worcester, MA 01655 USA Harvard Med Sch, Massachusetts Gen Hosp, Endocrine Unit, Boston, MA 02114 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USAChaugule, Sachin论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USAMa, Hong论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Microbiol & Physiol Syst, Worcester, MA 01655 USA UMass Chan Med Sch, Viral Vector Core, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USAXie, Jun论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Microbiol & Physiol Syst, Worcester, MA 01655 USA UMass Chan Med Sch, Viral Vector Core, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USAGao, Guangping论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Microbiol & Physiol Syst, Worcester, MA 01655 USA UMass Chan Med Sch, Viral Vector Core, Worcester, MA 01655 USA UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USAShim, Jae-Hyuck论文数: 0 引用数: 0 h-index: 0机构: UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USA UMass Chan Med Sch, Horae Gene Therapy Ctr, Worcester, MA 01655 USA UMass Chan Med Sch, Li Weibo Inst Rare Dis Res, Worcester, MA 01655 USA UMass Chan Med Sch, Dept Med, Div Rheumatol, Worcester, MA 01655 USA
- [2] AAV-mediated gene therapies by miniature gene editing toolsSCIENCE CHINA-LIFE SCIENCES, 2024, 67 (12) : 2540 - 2553Kong, Xiangfeng论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, Shanghai Inst Mat Med, Shanghai 201203, Peoples R China Chinese Acad Sci, Inst Neurosci, Ctr Excellence Brain Sci & Intelligence Technol, Shanghai 200031, Peoples R China Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201210, Peoples R ChinaLi, Tong论文数: 0 引用数: 0 h-index: 0机构: Chinese Acad Sci, Shanghai Inst Mat Med, Shanghai 201203, Peoples R China HuidaGene Therapeut Co Ltd, Shanghai 200131, Peoples R China Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201210, Peoples R ChinaYang, Hui论文数: 0 引用数: 0 h-index: 0机构: Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201210, Peoples R China Chinese Acad Sci, Shanghai Inst Mat Med, Shanghai 201203, Peoples R China Chinese Acad Sci, Inst Neurosci, Ctr Excellence Brain Sci & Intelligence Technol, Shanghai 200031, Peoples R China HuidaGene Therapeut Co Ltd, Shanghai 200131, Peoples R China Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201210, Peoples R China
- [3] AAV-mediated gene therapies by miniature gene editing toolsScience China(Life Sciences), 2024, 67 (12) : 2540 - 2553Xiangfeng Kong论文数: 0 引用数: 0 h-index: 0机构: Shanghai Institute of Materia Medica, Chinese Academy of Sciences Institute of Neuroscience, Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences Shanghai Institute of Materia Medica, Chinese Academy of SciencesTong Li论文数: 0 引用数: 0 h-index: 0机构: Shanghai Institute of Materia Medica, Chinese Academy of Sciences Huida Gene Therapeutics Co, Ltd Shanghai Institute of Materia Medica, Chinese Academy of SciencesHui Yang论文数: 0 引用数: 0 h-index: 0机构: Shanghai Center for Brain Science and Brain-Inspired Shanghai Institute of Materia Medica, Chinese Academy of Sciences Institute of Neuroscience, Center for Excellence in Brain Science and Intelligence Technology, Chinese Academy of Sciences Huida Gene Therapeutics Co, Ltd Shanghai Institute of Materia Medica, Chinese Academy of Sciences
- [4] AAV-mediated gene editing lights up the lungMOLECULAR THERAPY, 2022, 30 (01) : 7 - 9Yan, Ziying论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USALynch, Thomas J.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USAEngelhardt, John F.论文数: 0 引用数: 0 h-index: 0机构: Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USA Univ Iowa, Dept Anat & Cell Biol, Carver Coll Med, Room 1-100 BSB,51 Newton Rd, Iowa City, IA 52242 USA
- [5] AAV-mediated FOXG1 gene editing in human Rett primary cellsEUROPEAN JOURNAL OF HUMAN GENETICS, 2020, 28 (10) : 1446 - 1458Croci, Susanna论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, ItalyCarriero, Miriam Lucia论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, ItalyCapitani, Katia论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy ISPRO Core Res Lab CRL, Mol Mech Oncogenesis, Florence, Italy Univ Siena, Med Genet, Siena, ItalyDaga, Sergio论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, ItalyDonati, Francesco论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy ISPRO Core Res Lab CRL, Mol Mech Oncogenesis, Florence, Italy Univ Siena, Med Genet, Siena, Italy论文数: 引用数: h-index:机构:Frullanti, Elisa论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, ItalyLopergolo, Diego论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Azienda Osped Univ Senese, Genet Med, Siena, Italy Univ Siena, Med Genet, Siena, ItalyLamacchia, Vittoria论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Azienda Osped Univ Senese, Genet Med, Siena, Italy Univ Siena, Med Genet, Siena, ItalyTita, Rossella论文数: 0 引用数: 0 h-index: 0机构: Azienda Osped Univ Senese, Genet Med, Siena, Italy Univ Siena, Med Genet, Siena, ItalyGiliberti, Annarita论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, Italy论文数: 引用数: h-index:机构:Niccheri, Francesca论文数: 0 引用数: 0 h-index: 0机构: ISPRO Core Res Lab CRL, Mol Mech Oncogenesis, Florence, Italy Univ Siena, Med Genet, Siena, Italy论文数: 引用数: h-index:机构:Lo Rizzo, Caterina论文数: 0 引用数: 0 h-index: 0机构: Azienda Osped Univ Senese, Genet Med, Siena, Italy Univ Siena, Med Genet, Siena, ItalyConticello, Silvestro Giovanni论文数: 0 引用数: 0 h-index: 0机构: ISPRO Core Res Lab CRL, Mol Mech Oncogenesis, Florence, Italy Univ Siena, Med Genet, Siena, Italy论文数: 引用数: h-index:机构:Meloni, Ilaria论文数: 0 引用数: 0 h-index: 0机构: Univ Siena, Med Genet, Siena, Italy Univ Siena, Med Genet, Siena, Italy
- [6] AAV-mediated FOXG1 gene editing in human Rett primary cellsEuropean Journal of Human Genetics, 2020, 28 : 1446 - 1458Susanna Croci论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsMiriam Lucia Carriero论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsKatia Capitani论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsSergio Daga论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsFrancesco Donati论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsFilomena Tiziana Papa论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsElisa Frullanti论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsDiego Lopergolo论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsVittoria Lamacchia论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsRossella Tita论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsAnnarita Giliberti论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsElisa Benetti论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsFrancesca Niccheri论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsSimone Furini论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsCaterina Lo Rizzo论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsSilvestro Giovanni Conticello论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsAlessandra Renieri论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical GeneticsIlaria Meloni论文数: 0 引用数: 0 h-index: 0机构: University of Siena,Medical Genetics
- [7] 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
- [8] AAV-Mediated CRISPR/Cas Gene Editing of Retinal Cells In VivoINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2016, 57 (07) : 3470 - 3476Hung, Sandy S. C.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaChrysostomou, Vicki论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaLi, Fan论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Tasmania, Menzies Inst Med Res, Sch Med, Hobart, Tas, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaLim, Jeremiah K. H.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Dept Optometry & Vis Sci, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaWang, Jiang-Hui论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaPowell, Joseph E.论文数: 0 引用数: 0 h-index: 0机构: Univ Queensland, Inst Mol Biosci, Brisbane, Qld, Australia Univ Queensland, Queensland Brain Inst, Ctr Neurogenet & Stat Genom, Brisbane, Qld, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaTu, Leilei论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Jinan Univ, Dept Ophthalmol, Guangzhou, Guangdong, Peoples R China Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaDaniszewski, Maciej论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaLo, Camden论文数: 0 引用数: 0 h-index: 0机构: Monash Univ, Monash Micro Imaging, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaWong, Raymond C.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaCrowston, Jonathan G.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaPebay, Alice论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaKing, Anna E.论文数: 0 引用数: 0 h-index: 0机构: Univ Tasmania, Wicking Dementia Res & Educ Ctr, Hobart, Tas, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaBui, Bang V.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Dept Optometry & Vis Sci, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaLiu, Guei-Sheung论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, AustraliaHewitt, Alex W.论文数: 0 引用数: 0 h-index: 0机构: Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia Univ Tasmania, Menzies Inst Med Res, Sch Med, Hobart, Tas, Australia Univ Melbourne, Royal Victorian Eye & Ear Hosp, Ctr Eye Res Australia, Melbourne, Vic, Australia
- [9] 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
- [10] AAV-Mediated CRISPR/Cas9 Gene Editing in Murine PhenylketonuriaMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 234 - 245Richards, Daelyn Y.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USAWinn, Shelley R.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USADudley, Sandra论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USANygaard, Sean论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USAMighell, Taylor L.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA论文数: 引用数: h-index:机构:Harding, Cary O.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Pediat, Portland, OR 97239 USA Oregon Hlth & Sci Univ, Dept Mol & Med Genet, 3181 SW Sam Jackson Pk Rd,Mail Code L103, Portland, OR 97239 USA