AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease
about
Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's diseaseCAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroupDeveloping stem cell therapies for juvenile and adult-onset Huntington's diseaseEpigenetic mechanisms of neurodegeneration in Huntington's diseaseKnockdown and overexpression of NR1 modulates NMDA receptor function.The hematopoietic cytokine granulocyte-macrophage colony stimulating factor is important for cognitive functions.Glial promoter selectivity following AAV-delivery to the immature brain.Semaphorin 6A improves functional recovery in conjunction with motor training after cerebral ischemia.Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53-dependent myopathy in vivoEfficient allele-specific targeting of LRRK2 R1441 mutations mediated by RNAi.Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts.CNS-targeted viral delivery of G-CSF in an animal model for ALS: improved efficacy and preservation of the neuromuscular unitRNAi medicine for the brain: progresses and challenges.Gene therapy in mouse models of huntington disease.Ultrasensitive measurement of huntingtin protein in cerebrospinal fluid demonstrates increase with Huntington disease stage and decrease following brain huntingtin suppressionAAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancerIntestinal DMT1 is critical for iron absorption in the mouse but is not required for the absorption of copper or manganesesiRNA-mediated downregulation of GluN2B in the rostral anterior cingulate cortex attenuates mechanical allodynia and thermal hyperalgesia in a rat model of pain associated with bone cancer.Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease.Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics.Binge drinking upregulates accumbens mGluR5-Homer2-PI3K signaling: functional implications for alcoholism.KIBRA (KIdney/BRAin protein) regulates learning and memory and stabilizes Protein kinase Mζ.Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.Viral vector mediated expression of mutant huntingtin in the dorsal raphe produces disease-related neuropathology but not depressive-like behaviors in wildtype mice.Experimental surgical therapies for Huntington's disease.RNAi: a potential new class of therapeutic for human genetic disease.Pharmacologic approaches to the treatment of Huntington's disease.Using non-coding small RNAs to develop therapies for Huntington's disease.RNA therapy for polyglutamine neurodegenerative diseases.Spinocerebellar ataxia type 2: clinical presentation, molecular mechanisms, and therapeutic perspectives.Synthetic nucleic acids delivered by exosomes: a potential therapeutic for generelated metabolic brain diseases.New and improved AAVenues: current status of hemophilia B gene therapy.Astrocytes are key but indirect contributors to the development of the symptomatology and pathophysiology of Huntington's disease.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Recent Trends in Detection of Huntingtin and Preclinical Models of Huntington's DiseaseIntestinal brush-border Na+/H+ exchanger-3 drives H+-coupled iron absorption in the mouse.Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?
P2860
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P2860
AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease
description
2008 nî lūn-bûn
@nan
2008 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2008 թվականի մարտին հրատարակված գիտական հոդված
@hy
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
name
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@ast
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@en
type
label
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@ast
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@en
prefLabel
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@ast
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@en
P2093
P2860
P356
P1433
P1476
AAV vector-mediated RNAi of mu ...... model of Huntington's disease
@en
P2093
Dahna M Fong
Deborah Young
Helen L Fitzsimons
Matthew J During
Matthias Klugmann
Nicholas R Franich
P2860
P304
P356
10.1038/MT.2008.50
P577
2008-03-25T00:00:00Z