Zobrazeno 1 - 10 of 1 360 pro vyhledávání: '"Kozarsky K"'
1
Akademický článek
An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
Autor: Vasireddy V; SwanBio Therapeutics, Inc., Philadelphia, PA, USA., Maguire CA; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA., Anderson DW; SwanBio Therapeutics, Inc., Philadelphia, PA, USA., Ng C; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA., Gong Y; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA., Eichler F; Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.; Harvard Medical School, Boston, MA, USA., Fourcade S; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain., Guilera C; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain., Onieva A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Sanchez A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Leal-Julià M; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Verdés S; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain., Dijkstra IME; Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands., Kemp S; Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands., Park H; SwanBio Therapeutics, Inc., Philadelphia, PA, USA., Lutz T; SwanBio Therapeutics, Inc., Philadelphia, PA, USA., Clark SW; SwanBio Therapeutics, Inc., Philadelphia, PA, USA., Bosch A; Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.; Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.; Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain., Pujol A; Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.; Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.; Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain., Kozarsky K; SwanBio Therapeutics, Inc., Philadelphia, PA, USA.
Publikováno v: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Oct 09; Vol. 32 (4), pp. 101354. Date of Electronic Publication: 2024 Oct 09 (Print Publication: 2024).
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2
Akademický článek
In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord.
Autor: Hanlon KS; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA; University College London, London, UK., Cheng M; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Ferrer RM; Department of Pediatric Neurology, Emma Children's Hospital, Amsterdam UMC, Amsterdam Leukodystrophy Center, Amsterdam Neuroscience, University of Amsterdam, Amsterdam, the Netherlands; Department of Complex Trait Genetics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, Amsterdam Neuroscience, De Boelelaan, Amsterdam, the Netherlands., Ryu JR; Department of Anatomy, Brain Korea 21 Plus Program for Biomedical Science, Korea University College of Medicine, Seoul, Republic of Korea., Lee B; Department of Anatomy, Brain Korea 21 Plus Program for Biomedical Science, Korea University College of Medicine, Seoul, Republic of Korea., De La Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Patel N; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Espinoza P; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Santoscoy MC; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Gong Y; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Harvard Medical School, Boston, MA 02116, USA., Ng C; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Nguyen DM; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Nammour J; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA., Clark SW; SwanBio Therapeutics, Bala Cynwyd, PA 19005, USA., Heine VM; Department of Child and Adolescent Psychiatry, Emma Center for Personalized Medicine, Emma Children's Hospital, Amsterdam UMC, Amsterdam Neuroscience, Amsterdam, the Netherlands; Department of Complex Trait Genetics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, Amsterdam Neuroscience, De Boelelaan, Amsterdam, the Netherland., Sun W; Department of Anatomy, Brain Korea 21 Plus Program for Biomedical Science, Korea University College of Medicine, Seoul, Republic of Korea., Kozarsky K; SwanBio Therapeutics, Bala Cynwyd, PA 19005, USA., Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA 02115, USA; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA 02129, USA; Harvard Medical School, Boston, MA 02116, USA. Electronic address: cmaguire@mgh.harvard.edu.
Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Aug 07; Vol. 32 (8), pp. 2584-2603. Date of Electronic Publication: 2024 Jun 05.
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3
In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord.
Autor: Hanlon KS; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Cheng M; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., De La Cruz D; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Patel N; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Santoscoy MC; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Gong Y; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Harvard Medical School, Boston, MA., Ng C; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Nguyen DM; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Nammour J; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA., Clark SW; SwanBio Therapeutics, Bala Cynwyd, PA., Kozarsky K; SwanBio Therapeutics, Bala Cynwyd, PA., Maguire CA; Department of Neurology, Massachusetts General Hospital, Boston, MA.; Molecular Neurogenetics Unit, Massachusetts General Hospital, Charlestown, MA.; Harvard Medical School, Boston, MA.
Publikováno v: BioRxiv : the preprint server for biology [bioRxiv] 2023 Sep 13. Date of Electronic Publication: 2023 Sep 13.
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4
Akademický článek
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction.
Autor: Podetz-Pedersen KM; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Laoharawee K; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Singh S; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Nguyen TT; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Smith MC; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Temme A; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Kozarsky K; REGENXBIO, Inc., Rockville, Maryland, USA., McIvor RS; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA., Belur LR; Department of Genetics, Cell Biology and Development, Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA.
Publikováno v: Human gene therapy [Hum Gene Ther] 2023 Jan; Vol. 34 (1-2), pp. 8-18.
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5
Akademický článek
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6
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7
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8
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9
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10
Akademický článek
Nasal Drug Delivery and Nose-to-Brain Delivery Technology Development Status and Trend Analysis: Based on Questionnaire Survey and Patent Analysis.
Autor: Ge, Yuanyuan1,2 (AUTHOR) caomeng@smda.sh.cn, Xu, Xingying3 (AUTHOR) xyxu@libnet.sh.cn, Cao, Meng2 (AUTHOR) liaoping@smda.sh.cn, Liu, Baijun1 (AUTHOR) lbj1003@foxmail.com, Wang, Ying1 (AUTHOR) 15383267523@163.com, Liao, Ping2 (AUTHOR) wangjiajing@smda.sh.cn, Wang, Jiajing2 (AUTHOR) chenyifei25@smda.sh.cn, Chen, Yifei2 (AUTHOR), Yuan, Hongmei1 (AUTHOR) yuanhm612@163.com, Chen, Guiliang2 (AUTHOR) yuanhm612@163.com
Publikováno v: Pharmaceutics. Jul2024, Vol. 16 Issue 7, p929. 33p.
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