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