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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Delivery of genes to the brain and spinal cord across the blood-brain barrier (BBB) has not yet been achieved. Here we show that adeno-associated virus (AAV) 9 injected intravenously bypasses the BBBExpand
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Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo
Nat. Neurosci. 6, 21–27 (2003) During revision, panels were inadvertently exchanged in two figures. In Fig. 4, the panel labeled d should have been e and vice versa. In Fig. 5, the panel labeled bExpand
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Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model
Amyotrophic lateral sclerosis (ALS) is a progressive, lethal neuromuscular disease that is associated with the degeneration of spinal and brainstem motor neurons, leading to atrophy of limb, axial,Expand
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Astrocytes from Familial and Sporadic ALS Patients are Toxic to Motor Neurons
Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease, with astrocytes implicated as contributing substantially to motor neuron death in familial (F)ALS. However, the proposed role ofExpand
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The Adult Substantia Nigra Contains Progenitor Cells with Neurogenic Potential
In Parkinson's disease, progressive loss of dopaminergic neurons in the substantia nigra pars compacta (SN) leads to debilitating motor dysfunction. One current therapy aims at exogenous cellularExpand
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Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis
Neuroinflammation is one of the most striking hallmarks of amyotrophic lateral sclerosis (ALS). Nuclear factor-kappa B (NF-κB), a master regulator of inflammation, is upregulated in spinal cords ofExpand
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Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative disease affecting children, results in impaired motor neuron function. Despite knowledge of the pathogenic role ofExpand
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IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes
Adult multipotent neural progenitor cells can differentiate into neurons, astrocytes, and oligodendrocytes in the mammalian central nervous system, but the molecular mechanisms that control theirExpand
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A role for glia in the progression of Rett’s syndrome
Rett’s syndrome (RTT) is an X-chromosome-linked autism spectrum disorder caused by loss of function of the transcription factor methyl-CpG-binding protein 2 (MeCP2). Although MeCP2 is expressed inExpand
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Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy
Background Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or theExpand
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