Self-complementary AAV9 gene delivery partially corrects pathology associated with juvenile neuronal ceroid lipofuscinosis (CLN3)

Journal of Neuroscience

Volumn 36, Issue 37, 2016, Pages 9669-9682

  Bosch, Megan E ^a   Aldrich, Amy ^b   Fallet, Rachel ^b   Odvody, Jessica ^b   Burkovetskaya, Maria ^b   Schuberth, Kaitlyn ^a   Fitzgerald, Julie A ^c   Foust, Kevin D ^c   Kielian, Tammy ^b  

^a UNIVERSITY OF NEBRASKA MEDICAL CENTER   (United States)

^b UNIVERSITY OF NEBRASKA MEDICAL CENTER   (United States)

^c OHIO STATE UNIVERSITY   (United States)

Author keywords

AAV9; Astrocyte; Batten disease; CLN3; Lysosomal storage disease; Microglia

Indexed keywords

METHYL CPG BINDING PROTEIN 2; ACTIN; CHAPERONE; CLN3 PROTEIN, HUMAN; MEMBRANE PROTEIN;

ADENO ASSOCIATED VIRUS 9; ALLELE; ARTICLE; ASTROCYTE; BLOOD BRAIN BARRIER; BLOOD CHEMISTRY; CONTROLLED STUDY; DISEASE COURSE; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; HOMEOSTASIS; IMMUNOFLUORESCENCE TEST; LYSOSOME STORAGE DISEASE; MALE; MOUSE; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; REAL TIME POLYMERASE CHAIN REACTION; ROTAROD TEST; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; WESTERN BLOTTING; ANIMAL; BRAIN; C57BL MOUSE; COMPLICATION; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE TRANSFER; GENETICS; GLIA; HUMAN; METABOLISM; MOVEMENT DISORDERS; MUTATION; NERVE CELL; NEURONAL CEROID-LIPOFUSCINOSES; PATHOLOGY; TRANSGENIC MOUSE;

ACTINS; ANIMALS; BRAIN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; HUMANS; MALE; MEMBRANE GLYCOPROTEINS; METHYL-CPG-BINDING PROTEIN 2; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MOLECULAR CHAPERONES; MOVEMENT DISORDERS; MUTATION; NEUROGLIA; NEURONAL CEROID-LIPOFUSCINOSES; NEURONS;

EID: 84987792871     PISSN: 02706474     EISSN: 15292401     Source Type: Journal    
DOI: 10.1523/JNEUROSCI.1635-16.2016     Document Type: Article

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