Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
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Adeno-associated virus-mediated cancer gene therapy: current statusVision loss in juvenile neuronal ceroid lipofuscinosis (CLN3 disease)Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.Juvenile neuronal ceroid lipofuscinosis (Batten disease): current insightsPrevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar CellsLongitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease
P2860
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P2860
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
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2014 nî lūn-bûn
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2014 թուականի Մարտին հրատարակուած գիտական յօդուած
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2014 թվականի մարտին հրատարակված գիտական հոդված
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2014年の論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年论文
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name
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@ast
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@en
type
label
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@ast
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@en
prefLabel
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@ast
Partial correction of the CNS ...... xpressing the human CLN3 gene.
@en
P2093
P2860
P356
P1433
P1476
Partial correction of the CNS ...... expressing the human CLN3 gene
@en
P2093
Agnieszka Kubiak
Alvin Chen
Andrew M S Wong
Dolan Sondhi
Emma C Scott
Neil R Hackett
Ronald G Crystal
Susan L Cotman
Yewande Pearse
P2860
P304
P356
10.1089/HUM.2012.253
P577
2014-03-04T00:00:00Z