A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
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Biology of adeno-associated viral vectors in the central nervous systemRedirecting N-acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease.Increasing N-acetylaspartate in the Brain during Postnatal Myelination Does Not Cause the CNS Pathologies of Canavan Disease.Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice.Empty Virions In AAV8 Vector Preparations Reduce Transduction Efficiency And May Cause Total Viral Particle Dose-Limiting Side-EffectsLoss of central auditory processing in a mouse model of Canavan disease.Gene therapy for the nervous system: challenges and new strategies.Making the White Matter Matters: Progress in Understanding Canavan's Disease and Therapeutic Interventions Through Eight Decades.Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal miceClinical applications involving CNS gene transfer.In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene TherapyHost Anti-antibody Responses Following Adeno-associated Virus-mediated Delivery of Antibodies Against HIV and SIV in Rhesus MonkeysWidespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.Systemically administered AAV9-sTRAIL combats invasive glioblastoma in a patient-derived orthotopic xenograft model.rAAV Gene Therapy in a Canavan's Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System.Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes.Viral vectors for therapy of neurologic diseases.mRNA destabilization improves glycemic responsiveness of transcriptionally regulated hepatic insulin gene therapy in vitro and in vivo.N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase.Timing of Gene Therapy Interventions: The Earlier, the Better.Examination of the Blood Brain Barrier integrity in a mouse model of the neurodegenerative Canavan's disease.Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy.Brain Nat8l Knockdown Suppresses Spongiform Leukodystrophy in an Aspartoacylase-Deficient Canavan Disease Mouse Model.Genetic approaches to access cell types in mammalian nervous systems.Current Strategies for Brain Drug Delivery.A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.Leukodystrophies: Five new things.
P2860
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P2860
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
description
2013 nî lūn-bûn
@nan
2013 թուականի Յուլիսին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի հուլիսին հրատարակված գիտական հոդված
@hy
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
name
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@ast
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@en
type
label
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@ast
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@en
prefLabel
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@ast
A single intravenous rAAV inje ...... Gene therapy in Canavan mice.
@en
P2093
P2860
P356
P1433
P1476
A single intravenous rAAV inje ...... S Gene therapy in Canavan mice
@en
P2093
Ana A Liso Navarro
Andrew R Denninger
Chunyan Cao
Constance Moore
Elif M Sikoglu
Guangping Gao
Hongwei Zhang
Huapeng Li
P2860
P304
P356
10.1038/MT.2013.138
P577
2013-07-02T00:00:00Z