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- [1] Liver-Directed Gene Targeting as a Potential Therapy for Fabry DiseaseMOLECULAR THERAPY, 2024, 32 (04) : 733 - 733Saxena, Himanshi论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyBiasizzo, Jessica论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Inst Clin Pathol, Dept Lab Med, Udine, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyDomenis, Rossana论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Inst Clin Pathol, Dept Lab Med, Udine, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyZentilin, Lorena论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyDardis, Andrea论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Ctr Rare Dis, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyMuro, Andres F.论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, Italy
- [2] Liver-directed gene targeting as a potential therapy for Fabry diseaseHUMAN GENE THERAPY, 2022, 33 (23-24) : A170 - A170Saxena, H.论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyBiasizzo, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Inst Clin Pathol, Dept Lab Med, Udine, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyDomenis, R.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Inst Clin Pathol, Dept Lab Med, Udine, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyZentilin, L.论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyDardis, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Hosp Udine, Reg Coordinator Ctr Rare Dis, Udine, Italy Int Ctr Genet Engn & Biotechnol, Trieste, ItalyMuro, A. F.论文数: 0 引用数: 0 h-index: 0机构: Int Ctr Genet Engn & Biotechnol, Trieste, Italy Int Ctr Genet Engn & Biotechnol, Trieste, Italy
- [3] Liver-directed gene therapy corrects Fabry disease in miceMOLECULAR GENETICS AND METABOLISM, 2019, 126 (02) : S80 - S80Jeyakumar, Jey论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandKia, Azadeh论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandMcIntosh, Jenny论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandVerhoef, Daniel论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandKalcheva, Petya论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandHosseini, Paniz论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandSheridan, Rose论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandCorbau, Romuald论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England Freeline Therapeut, Stevenage, Herts, EnglandNathwani, Amit论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut, Stevenage, Herts, England UCL, Canc Inst, London, England Freeline Therapeut, Stevenage, Herts, England
- [4] Liver-directed gene therapyMEDICAL CLINICS OF NORTH AMERICA, 1996, 80 (05) : 1201 - &Nunes, FA论文数: 0 引用数: 0 h-index: 0机构: UNIV PENN,SCH MED,DIV GASTROENTEROL,PHILADELPHIA,PA 19104Raper, SE论文数: 0 引用数: 0 h-index: 0机构: UNIV PENN,SCH MED,DIV GASTROENTEROL,PHILADELPHIA,PA 19104
- [5] Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry diseaseGENE THERAPY, 2023, 30 (06) : 487 - 502Jeyakumar, Jey M.论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandKia, Azadeh论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandTam, Lawrence C. S.论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandMcIntosh, Jenny论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England Freeline Therapeut Ltd, Stevenage, EnglandSpiewak, Justyna论文数: 0 引用数: 0 h-index: 0机构: UCL, Inst Child Hlth, UCL Biol Mass Spectrometry Ctr, Genet & Genom Med Unit, London, England Freeline Therapeut Ltd, Stevenage, EnglandMills, Kevin论文数: 0 引用数: 0 h-index: 0机构: UCL, Inst Child Hlth, UCL Biol Mass Spectrometry Ctr, Genet & Genom Med Unit, London, England Freeline Therapeut Ltd, Stevenage, EnglandHeywood, Wendy论文数: 0 引用数: 0 h-index: 0机构: UCL, Inst Child Hlth, UCL Biol Mass Spectrometry Ctr, Genet & Genom Med Unit, London, England Freeline Therapeut Ltd, Stevenage, EnglandChisari, Elisa论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandCastaldo, Noemi论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandVerhoef, Daniel论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandHosseini, Paniz论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandKalcheva, Petya论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandCocita, Clement论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandMiranda, Carlos J.论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandCanavese, Miriam论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandKhinder, Jaminder论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandRosales, Cecilia论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England Freeline Therapeut Ltd, Stevenage, EnglandHughes, Derralynn论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England Royal Free London NHS Fdn Trust, London, England Freeline Therapeut Ltd, Stevenage, EnglandSheridan, Rose论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandCorbau, Romuald论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England Freeline Therapeut Ltd, Stevenage, EnglandNathwani, Amit论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, England UCL, London, England Royal Free London NHS Fdn Trust, London, England Freeline Therapeut Ltd, Stevenage, England
- [6] Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry diseaseGene Therapy, 2023, 30 : 487 - 502Jey M. Jeyakumar论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Azadeh Kia论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Lawrence C. S. Tam论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Jenny McIntosh论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Justyna Spiewak论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Kevin Mills论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Wendy Heywood论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Elisa Chisari论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Noemi Castaldo论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Daniël Verhoef论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Paniz Hosseini论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Petya Kalcheva论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Clement Cocita论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Carlos J. Miranda论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Miriam Canavese论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Jaminder Khinder论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Cecilia Rosales论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Derralynn Hughes论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Rose Sheridan论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Romuald Corbau论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,Amit Nathwani论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeutics,
- [7] Liver-Directed AAV Gene Therapy for Gaucher DiseaseBLOOD, 2019, 134Miranda, Carlos J.论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandCanavese, Miriam论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandChisari, Elisa论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandPandya, Jalpa论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandCocita, Clement论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandPortillo, Maria论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandMcIntosh, Jenny论文数: 0 引用数: 0 h-index: 0机构: UCL, Inst Canc, Dept Haematol, London, England Freeline, Stevenage, Herts, EnglandKia, Azadeh论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandFoley, Jonathan H.论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandDane, Allison论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandJeyakumar, Jey M.论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandSheridan, Rose论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandCorbau, Romuald论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England Freeline, Stevenage, Herts, EnglandNathwani, Amit C.论文数: 0 引用数: 0 h-index: 0机构: Freeline, Stevenage, Herts, England UCL, Inst Canc, Dept Haematol, London, England Freeline, Stevenage, Herts, England
- [8] Preclinical Development of ZS805, a Favorable Liver-Directed rAAV Gene Therapy for Fabry DiseaseMOLECULAR THERAPY, 2024, 32 (04) : 524 - 524Fan, Fuqin论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaHe, Siwu论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, Natl Clin Res Ctr Geriatr, Chengdu, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaZhang, Ming论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaLiu, Yu论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, Natl Clin Res Ctr Geriatr, Chengdu, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaZheng, Zhaoyue论文数: 0 引用数: 0 h-index: 0机构: Sichuan Univ, West China Hosp, Natl Clin Res Ctr Geriatr, Chengdu, Peoples R China Sichuan Univ, West China Hosp, State Key Lab Biotherapy, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaDong, Siyuan论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaGao, Lei论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaYe, Jingya论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R ChinaValencia, C. Alexander论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R China论文数: 引用数: h-index:机构:Dong, Biao论文数: 0 引用数: 0 h-index: 0机构: Sichuan Real & Best Biotech Co, Chengdu, Peoples R China Sichuan Real & Best Biotech Co, Chengdu, Peoples R China
- [9] Targeting the Apoa1 locus for liver-directed gene therapyMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 21 : 656 - 669De Giorgi, Marco论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USALi, Ang论文数: 0 引用数: 0 h-index: 0机构: Rice Univ, Dept Bioengn, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USAHurley, Ayrea论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USABarzi, Mercedes论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Dept Pediat, Div Med Genet, Durham, NC 27710 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USADoerfler, Alexandria M.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USACherayil, Nikitha A.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USASmith, Harrison E.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USABrown, Jonathan D.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Div Cardiovasc Med, Med Ctr, Nashville, TN 37232 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USALin, Charles Y.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA Baylor Coll Med, Therapeut Innovat Ctr, Dept Biochem & Mol Biol, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USABissig, Karl-Dimiter论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Dept Pediat, Div Med Genet, Durham, NC 27710 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USABao, Gang论文数: 0 引用数: 0 h-index: 0机构: Rice Univ, Dept Bioengn, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USALagor, William R.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USA Baylor Coll Med, Dept Mol Physiol & Biophys, Houston, TX 77030 USA
- [10] First-in-human study of a liver-directed AAV gene therapy (FLT190) in Fabry diseaseMOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S77 - S78Hughes, Derralynn A.论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, EnglandPatel, Niten论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandKinch, Russell论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandDronfield, Leanne论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandShort, Gerard论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandSheridan, Rose论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandKia, Azadeh论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandJeyakumar, Jey论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandCorbau, Romuald论文数: 0 引用数: 0 h-index: 0机构: Freeline Therapeut Ltd, Stevenage, Herts, England UCL, London, EnglandNathwani, Amit论文数: 0 引用数: 0 h-index: 0机构: UCL, London, England UCL, London, England