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  • Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.

    Author(s) : Serratrice, N.Cubizolle, A.Ibanes, S.Mestre-Francés, N.Bayo-Puxan, N.Creyssels, S.Gennetier, A.Bernex, F.Verdier, J. M.Haskins, M. E.Couderc, G.Malecaze, F.Kalatzis, V.Kremer, E. J.

    Author Affiliation : Institut de Génétique Moléculaire de Montpellier, CNRS 5535, 1919 Route de Mende, 34293 Montpellier, France.

    Author Email : eric.kremer@igmm.cnrs.fr

    Journal article : Journal of Controlled Release 2014 Vol.181 pp.22-31 ref.50

    Abstract : Corneal transparency is maintained, in part, by specialized fibroblasts called keratocytes, which reside in the fibrous lamellae of the stroma. Corneal clouding, a condition that impairs visual acuity, is associated with numerous diseases, including mucopolysaccharidosismucopolysaccharidosisSubject Category: Diseases, Disorders, and Symptoms
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    (MPS) type VII. MPS VII is due to deficiency in β-glucuronidase (β-glu) enzymatic activity, which leads to accumulation of glycosaminoglycansglycosaminoglycansSubject Category: Chemicals and Chemical Groups
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    (GAGs), and secondary accumulation of gangliosidesgangliosidesSubject Category: Chemicals and Chemical Groups
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    . Here, we tested the efficacy of canine adenoviruscanine adenovirusSubject Category: Organism Names
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    type 2 (CAV-2) vectors to transduce keratocyte in vivo in micemiceSubject Category: Organism Names
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    and nonhuman primatesprimatesSubject Category: Organism Names
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    , and ex vivo in dog and human corneal explants. Following efficacy studies, we asked if we could treat corneal clouding by the injection a helper-dependent (HD) CAV-2 vector (HD-RIGIE) harboring the human cDNA coding for β-glu (GUSB) in the canine MPS VII corneacorneaSubject Category: Anatomical and Morphological Structures
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    . β-Glu activity, GAG content, and lysosome morphology and physiopathologyphysiopathologySubject Category: Disciplines, Occupations and Industries
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    were analyzed. We found that HD-RIGIE injections efficiently transduced coxsackievirus adenovirus receptor-expressing keratocytes in the four species and, compared to mock-injected controls, improved the pathology in the canine MPS VII cornea. The key criterion to corrective therapy was the steady controlled release of β-glu and its diffusion throughout the collagencollagenSubject Category: Chemicals and Chemical Groups
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    -dense stroma. These data support the continued evaluation of HD CAV-2 vectors to treat diseases affecting corneal keratocytes.

    ISSN : 0168-3659

    DOI : 10.1016/j.jconrel.2014.02.022

    Record Number : 20143196835

    Publisher : Elsevier

    Location of publication : Amsterdam

    Country of publication : Netherlands

    Language of text : English

    Indexing terms for this abstract:

    Organism descriptor(s) : Canine adenoviruscanine adenovirusSubject Category: Organism Names
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    , dogsdogsSubject Category: Organism Names
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    , manmanSubject Category: Organism Names
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    , micemiceSubject Category: Organism Names
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    , PrimatesprimatesSubject Category: Organism Names
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    Descriptor(s) : beta-glucuronidasebeta-glucuronidaseSubject Category: Chemicals and Chemical Groups
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    , collagencollagenSubject Category: Chemicals and Chemical Groups
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    , corneacorneaSubject Category: Anatomical and Morphological Structures
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    , enzyme activityenzyme activitySubject Category: Properties
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    , enzymesenzymesSubject Category: Chemicals and Chemical Groups
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    , eyeseyesSubject Category: Anatomical and Morphological Structures
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    , gangliosidesgangliosidesSubject Category: Chemicals and Chemical Groups
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    , glycosaminoglycansglycosaminoglycansSubject Category: Chemicals and Chemical Groups
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    , lysosomeslysosomesSubject Category: Anatomical and Morphological Structures
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    , mucopolysaccharidosismucopolysaccharidosisSubject Category: Diseases, Disorders, and Symptoms
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    , physiopathologyphysiopathologySubject Category: Disciplines, Occupations and Industries
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    Identifier(s) : eye cornea, keratocytes, pathophysiology

    Broader term(s) : MastadenovirusmastadenovirusSubject Category: Organism Names
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    , AdenoviridaeadenoviridaeSubject Category: Organism Names
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    , dsDNA Virusesdsdna virusesSubject Category: Organism Names
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    , DNA Virusesdna virusesSubject Category: Organism Names
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    , virusesvirusesSubject Category: Organism Names
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    , CaniscanisSubject Category: Organism Names
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    , CanidaecanidaeSubject Category: Organism Names
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    , FissipedafissipedaSubject Category: Organism Names
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    , carnivorescarnivoresSubject Category: Organism Names
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    , mammalsmammalsSubject Category: Organism Names
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    , vertebratesvertebratesSubject Category: Organism Names
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    , ChordatachordataSubject Category: Organism Names
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    , animalsanimalsSubject Category: Organism Names
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    , eukaryoteseukaryotesSubject Category: Organism Names
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    , HomohomoSubject Category: Organism Names
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    , HominidaehominidaeSubject Category: Organism Names
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    , primatesprimatesSubject Category: Organism Names
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    , MuridaemuridaeSubject Category: Organism Names
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    , rodentsrodentsSubject Category: Organism Names
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