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    Tytuł:
    Improving retinal vascular endothelial cell tropism through rational rAAV capsid design.
    Autorzy:
    Periasamy R; Department of Ophthalmology and Visual Science, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.
    Patel DD; Department of Ophthalmology and Visual Science, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.; Cell Biology, Neurobiology, Anatomy, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.
    Boye SL; Department of Pediatrics, Powell Gene Therapy Center, University of Florida, Gainesville, FL, United States of America.
    Boye SE; Department of Pediatrics, Division of Cellular and Molecular Biology, University of Florida, Gainesville, FL, United States of America.
    Lipinski DM; Department of Ophthalmology and Visual Science, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.; Cell Biology, Neurobiology, Anatomy, Medical College of Wisconsin, Milwaukee, Wisconsin, United States of America.
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    Źródło:
    PloS one [PLoS One] 2023 May 11; Vol. 18 (5), pp. e0285370. Date of Electronic Publication: 2023 May 11 (Print Publication: 2023).
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural
    MeSH Terms:
    Capsid*/metabolism
    Dependovirus*/metabolism
    Mice ; Animals ; Cattle ; Humans ; Endothelial Cells ; Transduction, Genetic ; Genetic Therapy ; HEK293 Cells ; Genetic Vectors/genetics ; Retina/metabolism ; Capsid Proteins/genetics ; Capsid Proteins/metabolism ; Tropism
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS.
    Autorzy:
    Goodwin MS; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Croft CL; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Futch HS; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Ryu D; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Ceballos-Diaz C; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Liu X; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Paterno G; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Mejia C; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Deng D; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Menezes K; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Londono L; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Arjona K; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Parianos M; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Truong V; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Rostonics E; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Hernandez A; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Boye SL; Department of Pediatrics and the Powell Gene Therapy Center, University of Florida, Gainesville, Florida, USA.
    Boye SE; Department of Ophthalmology, University of Florida, Gainesville, Florida, USA.
    Levites Y; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA.; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA.; McKnight Brain Institute, College of Medicine, University of Florida, Gainesville, Florida, USA.
    Cruz PE; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA. .; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA. .
    Golde TE; Department of Neuroscience, College of Medicine, University of Florida, Gainesville, Florida, USA. .; Center for Translational Research in Neurodegenerative Disease, College of Medicine, University of Florida, Gainesville, Florida, USA. .; McKnight Brain Institute, College of Medicine, University of Florida, Gainesville, Florida, USA. .
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    Źródło:
    Molecular neurodegeneration [Mol Neurodegener] 2020 Mar 02; Vol. 15 (1), pp. 15. Date of Electronic Publication: 2020 Mar 02.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Dependovirus*
    Gene Expression*
    Gene Transfer Techniques*
    Genetic Vectors*
    Transduction, Genetic/*methods
    Animals ; Brain ; Genetic Therapy/methods ; Green Fluorescent Proteins/genetics ; HEK293 Cells ; Humans ; Mice ; Neuroglia ; Neurons
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknie Pełny tekstSzczegóły
    Tytuł:
    Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat.
    Autorzy:
    Bogner B; Department of Ophthalmology and Optometry, SALK/Paracelsus Medical University, Salzburg, Austria.
    Boye SL; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
    Min SH; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
    Peterson JJ; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
    Ruan Q; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
    Zhang Z; Department of Ophthalmology and Optometry, SALK/Paracelsus Medical University, Salzburg, Austria.
    Reitsamer HA; Department of Ophthalmology and Optometry, SALK/Paracelsus Medical University, Salzburg, Austria.
    Hauswirth WW; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
    Boye SE; Department of Ophthalmology, University of Florida, Gainesville, United States of America.
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    Źródło:
    PloS one [PLoS One] 2015 Jun 08; Vol. 10 (6), pp. e0128759. Date of Electronic Publication: 2015 Jun 08 (Print Publication: 2015).
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Transduction, Genetic*
    Anterior Chamber/*virology
    Capsid/*metabolism
    Dependovirus/*genetics
    Mutation/*genetics
    Trabecular Meshwork/*metabolism
    Animals ; Genetic Vectors ; Green Fluorescent Proteins ; Injections ; Mice, Inbred C57BL ; Rats, Sprague-Dawley
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.
    Autorzy:
    Boye SE; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America.
    Huang WC; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
    Roman AJ; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
    Sumaroka A; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
    Boye SL; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America.
    Ryals RC; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America.
    Olivares MB; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
    Ruan Q; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America.
    Tucker BA; Stephen A. Wynn Institute for Vision Research, University of Iowa Carver College of Medicine, Iowa City, Iowa, United States of America.
    Stone EM; Stephen A. Wynn Institute for Vision Research, University of Iowa Carver College of Medicine, Iowa City, Iowa, United States of America; Howard Hughes Medical Institute, University of Iowa Carver College of Medicine, Iowa City, Iowa, United States of America.
    Swaroop A; Neurobiology-Neurodegeneration & Repair Laboratory, National Eye Institute, National Institutes of Health, Bethesda, Maryland, United States of America.
    Cideciyan AV; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
    Hauswirth WW; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America.
    Jacobson SG; Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pennsylvania, United States of America.
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    Źródło:
    PloS one [PLoS One] 2014 Mar 26; Vol. 9 (3), pp. e92928. Date of Electronic Publication: 2014 Mar 26 (Print Publication: 2014).
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Disease Progression*
    Antigens, Neoplasm/*genetics
    Leber Congenital Amaurosis/*genetics
    Leber Congenital Amaurosis/*therapy
    Mutation/*genetics
    Neoplasm Proteins/*genetics
    Nuclear Proteins/*genetics
    Adolescent ; Adult ; Age Factors ; Animals ; Cell Cycle Proteins ; Child ; Cytoskeletal Proteins ; Disease Models, Animal ; Female ; Humans ; Leber Congenital Amaurosis/physiopathology ; Light Signal Transduction ; Mice, Inbred C57BL ; Mice, Transgenic ; Middle Aged ; Retina/pathology ; Retinal Cone Photoreceptor Cells/pathology ; Time Factors ; Tomography, Optical Coherence
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
    Autorzy:
    Lopes VS; Departments of Ophthalmology and Neurobiology, Jules Stein Eye Institute, UCLA School of Medicine, Los Angeles, CA 90095, USA.
    Boye SE
    Louie CM
    Boye S
    Dyka F
    Chiodo V
    Fofo H
    Hauswirth WW
    Williams DS
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    Źródło:
    Gene therapy [Gene Ther] 2013 Aug; Vol. 20 (8), pp. 824-33. Date of Electronic Publication: 2013 Jan 24.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Genetic Therapy*
    Myosins/*genetics
    Retinal Degeneration/*genetics
    Retinal Degeneration/*therapy
    Usher Syndromes/*therapy
    Animals ; DNA, Complementary ; Dependovirus ; Gene Expression ; Gene Transfer Techniques ; Green Fluorescent Proteins/genetics ; Humans ; Mice ; Myosin VIIa ; Myosins/metabolism ; Retina/pathology ; Retinal Degeneration/pathology ; Usher Syndromes/genetics ; Usher Syndromes/pathology
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors.
    Autorzy:
    Kay CN; Department of Ophthalmology, University of Florida College of Medicine, Gainesville, Florida, United States of America.
    Ryals RC
    Aslanidi GV
    Min SH
    Ruan Q
    Sun J
    Dyka FM
    Kasuga D
    Ayala AE
    Van Vliet K
    Agbandje-McKenna M
    Hauswirth WW
    Boye SL
    Boye SE
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    Źródło:
    PloS one [PLoS One] 2013 Apr 26; Vol. 8 (4), pp. e62097. Date of Electronic Publication: 2013 Apr 26 (Print Publication: 2013).
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Gene Transfer Techniques*
    Capsid/*metabolism
    Dependovirus/*genetics
    Genetic Vectors/*administration & dosage
    Mutation/*genetics
    Photoreceptor Cells, Vertebrate/*metabolism
    Animals ; Cell Line ; Chickens ; Dependovirus/physiology ; G-Protein-Coupled Receptor Kinase 1/genetics ; Gene Expression ; Humans ; Intravitreal Injections ; Mice ; Mice, Inbred C57BL ; Mice, Transgenic ; MicroRNAs/metabolism ; Promoter Regions, Genetic/genetics ; Serotyping ; Transduction, Genetic ; Transgenes/genetics ; Viral Tropism
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model.
    Autorzy:
    Jiang L; Department of Ophthalmology and Visual Sciences, University of Utah Health Science Center, Salt Lake City, Utah, USA. />Li TZ
    Boye SE
    Hauswirth WW
    Frederick JM
    Baehr W
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    Źródło:
    PloS one [PLoS One] 2013; Vol. 8 (3), pp. e57676. Date of Electronic Publication: 2013 Mar 05.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    RNA Interference*
    Guanylate Cyclase-Activating Proteins/*genetics
    Retinitis Pigmentosa/*genetics
    Alleles ; Animals ; Cloning, Molecular ; Dependovirus ; Disease Models, Animal ; Electroretinography ; Green Fluorescent Proteins/metabolism ; HEK293 Cells ; Humans ; Mice ; Mice, Transgenic ; Mutation ; RNA, Small Interfering/metabolism ; Retina/metabolism ; Retina/pathology ; Transgenes
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters.
    Autorzy:
    Beltran WA; Section of Ophthalmology, Department of Clinical Studies School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA. />Boye SL
    Boye SE
    Chiodo VA
    Lewin AS
    Hauswirth WW
    Aguirre GD
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    Źródło:
    Gene therapy [Gene Ther] 2010 Sep; Vol. 17 (9), pp. 1162-74. Date of Electronic Publication: 2010 Apr 29.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Promoter Regions, Genetic*
    Dependovirus/*genetics
    Genetic Therapy/*methods
    Retinal Rod Photoreceptor Cells/*metabolism
    Actins/genetics ; Actins/metabolism ; Animals ; Dogs ; G-Protein-Coupled Receptor Kinases/genetics ; G-Protein-Coupled Receptor Kinases/metabolism ; Genes, Reporter/genetics ; Genetic Vectors/genetics ; Humans ; Mice ; Transfection
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.
    Autorzy:
    Pang J; Department of Ophthalmology and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA. />Boye SE
    Lei B
    Boye SL
    Everhart D
    Ryals R
    Umino Y
    Rohrer B
    Alexander J
    Li J
    Dai X
    Li Q
    Chang B
    Barlow R
    Hauswirth WW
    Pokaż więcej
    Źródło:
    Gene therapy [Gene Ther] 2010 Jul; Vol. 17 (7), pp. 815-26. Date of Electronic Publication: 2010 Mar 18.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
    MeSH Terms:
    Genetic Therapy*
    Carrier Proteins/*physiology
    Dependovirus/*genetics
    Eye Proteins/*physiology
    Optic Atrophy, Hereditary, Leber/*therapy
    Retinal Cone Photoreceptor Cells/*physiology
    Retinal Degeneration/*genetics
    Retinal Degeneration/*prevention & control
    Animals ; Disease Models, Animal ; Genetic Vectors ; Mice ; cis-trans-Isomerases
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
    Tytuł:
    Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.
    Autorzy:
    Boye SE; Department of Ophthalmology, College of Medicine, University of Florida, Gainesville, Florida, United States of America. />Boye SL
    Pang J
    Ryals R
    Everhart D
    Umino Y
    Neeley AW
    Besharse J
    Barlow R
    Hauswirth WW
    Pokaż więcej
    Źródło:
    PloS one [PLoS One] 2010 Jun 25; Vol. 5 (6), pp. e11306. Date of Electronic Publication: 2010 Jun 25.
    Typ publikacji:
    Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
    MeSH Terms:
    Genetic Therapy*
    Guanylate Cyclase/*genetics
    Receptors, Cell Surface/*genetics
    Vision Disorders/*therapy
    Animals ; Base Sequence ; Behavior, Animal ; DNA Primers ; Dependovirus/genetics ; Electroretinography ; Genetic Vectors ; Immunohistochemistry ; Mice ; Mice, Knockout ; Photoreceptor Cells, Vertebrate/physiology ; Polymerase Chain Reaction ; Transgenes ; Vision Disorders/physiopathology ; Visual Acuity
    Czasopismo naukowe
    Pełny tekst  Link otwiera się w nowym oknieSzczegóły
      Wyświetlanie 1-10 z 10

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