Zobrazeno 1 - 10
of 168 277
pro vyhledávání: '"Genetic vectors"'
Autor:
de Moura Gomes A; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., L Petkau T; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Polymorphic BioSciences, Vancouver, BC, Canada., J Korecki A; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., Fornes O; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Department of Medical Genetics, The University of British Columbia, Vancouver, V6T 1Z3, Canada.; Genentech, South San Francisco, CA, USA., Galvan A; Udall Center of Excellence for Parkinson's Disease and Department of Neurology, Emory National Primate Research Center, Emory University, Atlanta, GA, 30329, USA., Lu G; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., M Hill A; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., Ling Lam S; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., Yao A; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., A Farkas R; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada., W Wasserman W; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Department of Medical Genetics, The University of British Columbia, Vancouver, V6T 1Z3, Canada., Smith Y; Udall Center of Excellence for Parkinson's Disease and Department of Neurology, Emory National Primate Research Center, Emory University, Atlanta, GA, 30329, USA., M Simpson E; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada.; Department of Medical Genetics, The University of British Columbia, Vancouver, V6T 1Z3, Canada., R Leavitt B; Centre for Molecular Medicine and Therapeutics at British Columbia Children's Hospital, The University of British Columbia, 2028-950 West 28th Avenue, Vancouver, BC, V5Z 4H4, Canada. bleavitt@cmmt.ubc.ca.; The Djavad Mowafaghian Center for Brain Health, The University of British Columbia, Vancouver, BC, V6T 1Z3, Canada. bleavitt@cmmt.ubc.ca.; Division of Neurology, Department of Medicine, University of British Columbia Hospital, Vancouver, BC, V6T 2B5, Canada. bleavitt@cmmt.ubc.ca.; Department of Medical Genetics, The University of British Columbia, Vancouver, V6T 1Z3, Canada. bleavitt@cmmt.ubc.ca.
Publikováno v:
Scientific reports [Sci Rep] 2024 Nov 15; Vol. 14 (1), pp. 28194. Date of Electronic Publication: 2024 Nov 15.
Autor:
Giaccio M; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy., Monaco A; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy., Galiano L; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy., Parente A; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy; Dipartimento di Scienze Mediche Traslazionali, Università Degli Studi di Napoli 'Federico II' Via S. Pansini, 5, Napoli, Italy., Borzacchiello L; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy; Dipartimento di Scienze Mediche Traslazionali, Università Degli Studi di Napoli 'Federico II' Via S. Pansini, 5, Napoli, Italy., Rubino R; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy., Klärner FG; Department of Chemistry, University of Duisburg-Essen, Universitaetsstrasse 7, 45117 Essen, Germany., Killa D; Department of Chemistry, University of Duisburg-Essen, Universitaetsstrasse 7, 45117 Essen, Germany., Perna C; Telethon Institute of Genetics and Medicine (TIGEM), Via C. Flegrei, 34, Pozzuoli, Napoli, Italy., Piccolo P; Telethon Institute of Genetics and Medicine (TIGEM), Via C. Flegrei, 34, Pozzuoli, Napoli, Italy., Marotta M; Dipartimento di Medicina Clinica e Chirurgia, Università Degli Studi di Napoli 'Federico II' Via S. Pansini, 5, Napoli, Italy., Pan X; Department of Pediatrics, Centre Hospitalier Universitaire Sainte-Justine Research Center, University of Montreal, Montreal, QC, Canada., Khijniak M; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA., Siddique I; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA., Schrader T; Department of Chemistry, University of Duisburg-Essen, Universitaetsstrasse 7, 45117 Essen, Germany., Pshezhetsky AV; Department of Pediatrics, Centre Hospitalier Universitaire Sainte-Justine Research Center, University of Montreal, Montreal, QC, Canada., Sorrentino NC; Telethon Institute of Genetics and Medicine (TIGEM), Via C. Flegrei, 34, Pozzuoli, Napoli, Italy; Dipartimento di Medicina Clinica e Chirurgia, Università Degli Studi di Napoli 'Federico II' Via S. Pansini, 5, Napoli, Italy., Bitan G; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; Brain Research Institute and Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA, USA., Fraldi A; CEINGE-Biotecnologie Avanzate Franco Salvatore, Via G. Salvatore, 486 Napoli, Italy; Dipartimento di Medicina Clinica e Chirurgia, Università Degli Studi di Napoli 'Federico II' Via S. Pansini, 5, Napoli, Italy. Electronic address: alessandro.fraldi@unina.it.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Nov 06; Vol. 32 (11), pp. 4108-4121. Date of Electronic Publication: 2024 Sep 28.
Autor:
Yoon JK; AVROBIO, Inc., Cambridge, MA 02139, USA., Schindler JW; AVROBIO, Inc., Cambridge, MA 02139, USA., Loperfido M; AVROBIO, Inc., Cambridge, MA 02139, USA., Baricordi C; AVROBIO, Inc., Cambridge, MA 02139, USA., DeAndrade MP; AVROBIO, Inc., Cambridge, MA 02139, USA., Jacobs ME; AVROBIO, Inc., Cambridge, MA 02139, USA., Treleaven C; AVROBIO, Inc., Cambridge, MA 02139, USA., Plasschaert RN; AVROBIO, Inc., Cambridge, MA 02139, USA., Yan A; AVROBIO, Inc., Cambridge, MA 02139, USA., Barese CN; AVROBIO, Inc., Cambridge, MA 02139, USA., Dogan Y; AVROBIO, Inc., Cambridge, MA 02139, USA., Chen VP; AVROBIO, Inc., Cambridge, MA 02139, USA., Fiorini C; AVROBIO, Inc., Cambridge, MA 02139, USA., Hull F; AVROBIO, Inc., Cambridge, MA 02139, USA., Barbarossa L; AVROBIO, Inc., Cambridge, MA 02139, USA., Unnisa Z; AVROBIO, Inc., Cambridge, MA 02139, USA., Ivanov D; AVROBIO, Inc., Cambridge, MA 02139, USA., Kutner RH; AVROBIO, Inc., Cambridge, MA 02139, USA., Guda S; AVROBIO, Inc., Cambridge, MA 02139, USA., Oborski C; AVROBIO, Inc., Cambridge, MA 02139, USA., Maiwald T; AVROBIO, Inc., Cambridge, MA 02139, USA., Michaud V; Lady Davis Institute for Medical Research, Jewish General Hospital, McGill University, Montreal, Quebec H3T 1E2, Canada., Rothe M; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Pfeifer R; AVROBIO, Inc., Cambridge, MA 02139, USA., Mason C; AVROBIO, Inc., Cambridge, MA 02139, USA; Advanced Centre for Biochemical Engineering, University College London, London WC1E 6AE, UK., Biasco L; AVROBIO, Inc., Cambridge, MA 02139, USA; Zayed Centre for Research, University College London, London WC1N 1DZ, UK., van Til NP; AVROBIO, Inc., Cambridge, MA 02139, USA; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Center, VU University, and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1081 HV, Amsterdam, the Netherlands; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081 HV, Amsterdam, the Netherlands. Electronic address: n.p.vantil@amsterdamumc.nl.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Nov 06; Vol. 32 (11), pp. 3847-3864. Date of Electronic Publication: 2024 Sep 17.
Autor:
Pham V; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Tricoli L; Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Hong X; Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Wongkittichote P; Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok 10400, Thailand., Castruccio Castracani C; Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Guerra A; Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Schlotawa L; Department of Pediatrics and Adolescent Medicine, University Medical Center Goettingen, 37075 Goettingen, Germany; Translational Neuroinflammation and Automated Microscopy, Fraunhofer Institute for Translational Medicine and Pharmacology, 37075 Goettingen, Germany., Adang LA; Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Kuhs A; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Cassidy MM; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Kane O; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Tsai E; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA., Presa M; The Jackson Laboratory, Rare Disease Translational Center, Bar Harbor, ME 04609, USA., Lutz C; The Jackson Laboratory, Rare Disease Translational Center, Bar Harbor, ME 04609, USA., Rivella SB; Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Institute for Regenerative Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; RNA Institute, University of Pennsylvania, Philadelphia, PA 19104, USA., Ahrens-Nicklas RC; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA; Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: ahrensnicklasr@email.chop.edu.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Nov 06; Vol. 32 (11), pp. 3829-3846. Date of Electronic Publication: 2024 Aug 22.
Autor:
Sun YD; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China., Li GH; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China., Zhang F; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China., Cheng T; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China., Zhang JP; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China., Zhang XB; State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, People's Republic of China.; Tianjin Institutes of Health Science, Tianjin 301600, People's Republic of China.
Publikováno v:
Stem cells (Dayton, Ohio) [Stem Cells] 2024 Nov 05; Vol. 42 (11), pp. 992-1005.
Autor:
Karan KR; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA., Andrzejewski S; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA., Stiles KM; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA., Hackett NR; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA., Crystal RG; Department of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2024 Nov; Vol. 35 (21-22), pp. 904-916. Date of Electronic Publication: 2024 Oct 16.
Autor:
Daci R; Department of Neurosurgery, UMass Chan Medical School, Worcester, Massachusetts, USA.; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, Massachusetts, USA., Gray-Edwards H; Department of Radiology, UMass Chan Medical School, Worcester, Massachusetts, USA.; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, Massachusetts, USA., Shazeeb MS; Department of Radiology, UMass Chan Medical School, Worcester, Massachusetts, USA., Vardar Z; Department of Radiology, UMass Chan Medical School, Worcester, Massachusetts, USA., Vachha B; Department of Radiology, UMass Chan Medical School, Worcester, Massachusetts, USA., Cataltepe OI; Department of Neurosurgery, UMass Chan Medical School, Worcester, Massachusetts, USA., Flotte TR; Department of Pediatrics, UMass Chan Medical School, Worcester, Massachusetts, USA.; Horae Gene Therapy Center, UMass Chan Medical School, Worcester, Massachusetts, USA.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2024 Nov; Vol. 35 (21-22), pp. 886-895. Date of Electronic Publication: 2024 Nov 08.
Autor:
Zhang F; School of Biotechnology, East China University of Science and Technology, Shanghai, China., Zhou X; School of Pharmacy, East China University of Science and Technology, Shanghai, China., Hua B; Department of Hematology, Beijing Shijitan Hospital, Capital Medical University, Beijing, China., He X; School of Pharmacy, East China University of Science and Technology, Shanghai, China., Li Z; School of Pharmacy, East China University of Science and Technology, Shanghai, China., Xiao X; School of Biotechnology, East China University of Science and Technology, Shanghai, China.; School of Pharmacy, East China University of Science and Technology, Shanghai, China., Wu X; School of Pharmacy, East China University of Science and Technology, Shanghai, China. wuxia@ecust.edu.cn.
Publikováno v:
Gene therapy [Gene Ther] 2024 Nov; Vol. 31 (11-12), pp. 544-552. Date of Electronic Publication: 2024 Sep 10.
Autor:
Lu Q; Department of Ophthalmology, Visual and Anatomical Sciences, Wayne State University School of Medicine, Detroit, MI, USA., Wright A; Department of Ophthalmology, Visual and Anatomical Sciences, Wayne State University School of Medicine, Detroit, MI, USA., Pan ZH; Department of Ophthalmology, Visual and Anatomical Sciences, Wayne State University School of Medicine, Detroit, MI, USA. zhpan@med.wayne.edu.
Publikováno v:
Gene therapy [Gene Ther] 2024 Nov; Vol. 31 (11-12), pp. 572-579. Date of Electronic Publication: 2024 Sep 05.
Autor:
McCarron A; Adelaide Medical School, The University of Adelaide, Adelaide, SA, Australia. alexandra.mccarron@adelaide.edu.au.; Robinson Research Institute, The University of Adelaide, Adelaide, SA, Australia. alexandra.mccarron@adelaide.edu.au.; Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, SA, Australia. alexandra.mccarron@adelaide.edu.au., Ling KM; Wal-Yan Respiratory Research Centre, Telethon Kids Institute, Nedlands, WA, Australia.; School of Population Health, Curtin University, Bentley, WA, Australia., Montgomery ST; Wal-Yan Respiratory Research Centre, Telethon Kids Institute, Nedlands, WA, Australia.; School of Population Health, Curtin University, Bentley, WA, Australia., Martinovich KM; Wal-Yan Respiratory Research Centre, Telethon Kids Institute, Nedlands, WA, Australia.; Centre for Child Health Research, University of Western Australia, Crawley, WA, Australia., Cmielewski P; Adelaide Medical School, The University of Adelaide, Adelaide, SA, Australia.; Robinson Research Institute, The University of Adelaide, Adelaide, SA, Australia.; Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, SA, Australia., Rout-Pitt N; Adelaide Medical School, The University of Adelaide, Adelaide, SA, Australia.; Robinson Research Institute, The University of Adelaide, Adelaide, SA, Australia.; Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, SA, Australia., Kicic A; Wal-Yan Respiratory Research Centre, Telethon Kids Institute, Nedlands, WA, Australia.; School of Population Health, Curtin University, Bentley, WA, Australia.; Department of Respiratory and Sleep, Perth Children's Hospital, Nedlands, WA, Australia.; Centre for Cell Therapy and Regenerative Medicine, School of Medicine and Pharmacology, The University of Western Australia and Harry Perkins Institute of Medical Research, Nedlands, WA, Australia., Parsons D; Adelaide Medical School, The University of Adelaide, Adelaide, SA, Australia.; Robinson Research Institute, The University of Adelaide, Adelaide, SA, Australia.; Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, SA, Australia., Donnelley M; Adelaide Medical School, The University of Adelaide, Adelaide, SA, Australia.; Robinson Research Institute, The University of Adelaide, Adelaide, SA, Australia.; Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, SA, Australia.
Publikováno v:
Gene therapy [Gene Ther] 2024 Nov; Vol. 31 (11-12), pp. 553-559. Date of Electronic Publication: 2024 Aug 25.