Gene Therapy for Neurodegenerative Diseases
نویسندگان
چکیده
منابع مشابه
Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors.
Gene therapy holds great promise for the treatment of a wide range of inherited and acquired disorders. The development of viral vector systems to mediate safe and long-lasting expression of therapeutic transgenes in specific target cell populations is continually advancing. Gene therapy for the nervous system is particularly challenging due to the post-mitotic nature of neuronal cells and the ...
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002 drugs against AD [7]. Bone marrow-derived mesenchymal stem cells (MSCs) were able to home on the injured brain and remove Abeta plaques from the hippocampus and to reduce Abeta deposits through the activation of endogenous microglia in an induced AD mouse model [8]. Human umbilical cord-derived MSCs have been shown to improve memory deficits, and reduce Abeta deposition [9]. Moreover, one r...
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Neurodegenerative diseases, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), Parkinson’s disease (PD), frontotemporal lobar degeneration (FTLD), Huntington’s disease (HD), and multiple sclerosis (MS), are characterized by progressive degeneration of particular neuronal cells. Neurodegenerative disorders are believed to share a common pathophysiologic mechanism that allows the depo...
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Diseases resulting from defects in a single gene may be more amenable to treatment by conventional gene therapy strategies than idiopathic or polygenic disorders. We have attempted to reduce the expression in vivo of the Huntington's disease gene protein, Huntingtin, using an 18-mer fluorescein-labeled phosphorothiorated antisense oligodeoxynucleotide (ODN) targeted against the start site of th...
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Since the first reports that double-stranded RNAs can efficiently silence gene expression in C. elegans, the technology of RNA interference (RNAi) has been intensively exploited as an experimental tool to study gene function. With the subsequent discovery that RNAi could also be applied to mammalian cells, the technology of RNAi expanded from being a valuable experimental tool to being an appli...
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ژورنال
عنوان ژورنال: Neurotherapeutics
سال: 2018
ISSN: 1933-7213,1878-7479
DOI: 10.1007/s13311-018-00694-0