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Department of Neuroscience
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Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.
Molecular therapy : the journal of the American Society of Gene Therapy Mar, 2006 | Pubmed ID: 16412695
Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons.
Human gene therapy Jan, 2008 | Pubmed ID: 18052722
Recombinant adeno-associated virus-mediated global anterograde delivery of glial cell line-derived neurotrophic factor to the spinal cord: comparison of rubrospinal and corticospinal tracts in the rat.
Human gene therapy Jan, 2008 | Pubmed ID: 18072858
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.
Nature biotechnology Jan, 2009 | Pubmed ID: 19098898
Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity.
Molecular therapy : the journal of the American Society of Gene Therapy Jun, 2009 | Pubmed ID: 19277011
AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS.
Current gene therapy Oct, 2009 | Pubmed ID: 19860657
Over the barrier and through the blood: to CNS delivery we go.
Cell cycle (Georgetown, Tex.) Dec, 2009 | Pubmed ID: 19949299
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
Nature biotechnology Mar, 2010 | Pubmed ID: 20190738
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.
Human molecular genetics Oct, 2010 | Pubmed ID: 20639395
A role for glia in the progression of Rett's syndrome.
Nature Jul, 2011 | Pubmed ID: 21716289
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.
Molecular therapy : the journal of the American Society of Gene Therapy Nov, 2011 | Pubmed ID: 21811247
Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.
Nature biotechnology Sep, 2011 | Pubmed ID: 21832997
Recombinant AAV delivery to the central nervous system.
Methods in molecular biology (Clifton, N.J.) , 2011 | Pubmed ID: 22034030
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.
Human molecular genetics Apr, 2012 | Pubmed ID: 22186025
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates.
Human gene therapy Apr, 2012 | Pubmed ID: 22201473
Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound.
Human gene therapy Nov, 2012 | Pubmed ID: 22838844
Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome.
The Journal of neuroscience : the official journal of the Society for Neuroscience Aug, 2013 | Pubmed ID: 23966684
Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.
Molecular therapy : the journal of the American Society of Gene Therapy Dec, 2013 | Pubmed ID: 24008656
Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis.
Neuron Mar, 2014 | Pubmed ID: 24607225
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA - a dose response study in mice and nonhuman primates.
Molecular therapy : the journal of the American Society of Gene Therapy Oct, 2014 | Pubmed ID: 25358252
Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice.
Frontiers in molecular neuroscience , 2014 | Pubmed ID: 25360081
Ohio State University
Sara E. Gombash Lampe1,
Brian K. Kaspar2,
Kevin D. Foust1
1Department of Neuroscience, Ohio State University,
2Center for Gene Therapy, Nationwide Children's Hospital Research Institute, Ohio State University
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