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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS
M. Bravo-Hernandez, T. Tadokoro, MR. Navarro, O. Platoshyn, Y. Kobayashi, S. Marsala, A. Miyanohara, S. Juhas, J. Juhasova, H. Skalnikova, Z. Tomori, I. Vanicky, H. Studenovska, V. Proks, P. Chen, N. Govea-Perez, D. Ditsworth, JD. Ciacci, S. Gao,...
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, Research Support, N.I.H., Extramural, práce podpořená grantem
Grantová podpora
R01 CA134633
NCI NIH HHS - United States
NLK
ProQuest Central
od 2000-01-01 do Před 1 rokem
Health & Medicine (ProQuest)
od 2000-01-01 do Před 1 rokem
- MeSH
- amyotrofická laterální skleróza genetika patofyziologie terapie MeSH
- atrofie MeSH
- degenerace nervu genetika patofyziologie terapie MeSH
- Dependovirus metabolismus MeSH
- interneurony patologie MeSH
- lidé MeSH
- malá interferující RNA aplikace a dávkování MeSH
- messenger RNA genetika metabolismus MeSH
- mícha diagnostické zobrazování patologie patofyziologie MeSH
- motorické evokované potenciály MeSH
- motorické neurony patologie MeSH
- myši inbrední C57BL MeSH
- myši transgenní MeSH
- pia mater patologie patofyziologie MeSH
- prasata MeSH
- primáti MeSH
- progrese nemoci MeSH
- regulace genové exprese MeSH
- sbalování proteinů MeSH
- superoxid dismutáza 1 genetika metabolismus MeSH
- technika přenosu genů * MeSH
- umlčování genů * MeSH
- vývoj svalů MeSH
- zánět patologie MeSH
- zvířata MeSH
- Check Tag
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- zvířata MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- Research Support, N.I.H., Extramural MeSH
Gene silencing with virally delivered shRNA represents a promising approach for treatment of inherited neurodegenerative disorders. In the present study we develop a subpial technique, which we show in adult animals successfully delivers adeno-associated virus (AAV) throughout the cervical, thoracic and lumbar spinal cord, as well as brain motor centers. One-time injection at cervical and lumbar levels just before disease onset in mice expressing a familial amyotrophic lateral sclerosis (ALS)-causing mutant SOD1 produces long-term suppression of motoneuron disease, including near-complete preservation of spinal α-motoneurons and muscle innervation. Treatment after disease onset potently blocks progression of disease and further α-motoneuron degeneration. A single subpial AAV9 injection in adult pigs or non-human primates using a newly designed device produces homogeneous delivery throughout the cervical spinal cord white and gray matter and brain motor centers. Thus, spinal subpial delivery in adult animals is highly effective for AAV-mediated gene delivery throughout the spinal cord and supraspinal motor centers.
Department of Neurosurgery University of California San Diego La Jolla CA USA
Department of Radiology University of California San Diego La Jolla CA USA
Dept of Biophysics Institute of Experimental Physics Slovak Academy of Sciences Kosice Slovakia
Institute of Animal Physiology and Genetics AS CR v v i Liběchov Czech Republic
Institute of Neurobiology Slovak Academy of Sciences Kosice Slovakia
Citace poskytuje Crossref.org
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