Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9â•’ADAR2 delivery to motor neurons
نویسندگان
چکیده
(1) CREST, Japan Science and Technology Agency, Medicine, University of Tokyo, Bunkyo-Ku, Tokyo, Ja (2) Department of Neurology, Graduate School of Me Tokyo, Bunkyo-Ku, Tokyo, Japan (3) Center for Disease Biology and Integrative Medicine Medicine, The University of Tokyo, Bunkyo-Ku, Toky (4) Department of Neurosurgery, Jichi Medical Uni Tochigi, Japan (5) Department of Neurology, Jichi Medical University, Japan (6) Clinical Research Center for Medicine, International and Welfare, Ichikawa, Chiba, Japan *Corresponding author: Tel: þ81 3 5841 3566; Fax: þ E-mail: [email protected]
منابع مشابه
Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons
Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease, and the lack of effective therapy results in inevitable death within a few years of onset. Failure of GluA2 RNA editing resulting from downregulation of the RNA-editing enzyme adenosine deaminase acting on RNA 2 (ADAR2) occurs in the majority of ALS cases and causes the death of motor neurons via a Ca(2+) -...
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Transactive response DNA-binding protein (TDP-43) pathology, and failure of A-to-I conversion (RNA editing) at the glutamine/arginine (Q/R) site of α-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid (AMPA) receptor subunit GluA2, are etiology-linked molecular abnormalities that concomitantly occur in the motor neurons of most patients with amyotrophic lateral sclerosis (ALS). Adenosine deami...
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Amyotrophic lateral sclerosis (ALS) is a common adult-onset nervous system degenerative disease, characterized by the progressive loss of upper and lower motor neurons. TDP-43 pathology in motor neurons is a hallmark of ALS. In addition, the reduced expression of an RNAediting enzyme, adenosine deaminase acting on RNA 2 (ADAR2), increases the expression of GluA2 at an unedited glutamine/arginin...
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Nuclear dysfunction in motor neurons has been hypothesized to be a principal cause of amyotrophic lateral sclerosis (ALS) pathogenesis. Here, we investigated the mechanism by which the nuclear pore complex (NPC) is disrupted in dying motor neurons in a mechanistic ALS mouse model (adenosine deaminase acting on RNA 2 (ADAR2) conditional knockout (AR2) mice) and in ALS patients. We showed that nu...
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TDP-43 pathology in spinal motor neurons is a neuropathological hallmark of sporadic amyotrophic lateral sclerosis (ALS) and has recently been shown to be closely associated with the downregulation of an RNA editing enzyme called adenosine deaminase acting on RNA 2 (ADAR2) in the motor neurons of sporadic ALS patients. Because TDP-43 pathology is found more frequently in the brains of elderly p...
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تاریخ انتشار 2013