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Nielsen TT, Nielsen JE. Antisense gene silencing: therapy for neurodegenerative disorders?. Genes (Basel). 2013;4(3):457-84doi: 10.3390/genes4030457.
Nielsen, T. T., & Nielsen, J. E. (2013). Antisense gene silencing: therapy for neurodegenerative disorders?. Genes, 4(3), 457-84. https://doi.org/10.3390/genes4030457
Nielsen, Troels T, and Nielsen, Jørgen E. "Antisense gene silencing: therapy for neurodegenerative disorders?." Genes vol. 4,3 (2013): 457-84. doi: https://doi.org/10.3390/genes4030457
Nielsen TT, Nielsen JE. Antisense gene silencing: therapy for neurodegenerative disorders?. Genes (Basel). 2013 Sep 10;4(3):457-84. doi: 10.3390/genes4030457. PMID: 24705213; PMCID: PMC3924827.
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Genes (Basel). 2013 Sep 10;4(3):457-84. doi: 10.3390/genes4030457.

Antisense gene silencing: therapy for neurodegenerative disorders?.

Genes

Troels T Nielsen, Jørgen E Nielsen

Affiliations

  1. Danish Dementia Research Centre, Neurogenetics Clinic, Department of Neurology, Section 6702, Rigshospitalet, Copenhagen University Hospital, Blegdamsvej 9, DK-2100, Copenhagen Ø, Denmark. [email protected].
  2. Danish Dementia Research Centre, Neurogenetics Clinic, Department of Neurology, Section 6702, Rigshospitalet, Copenhagen University Hospital, Blegdamsvej 9, DK-2100, Copenhagen Ø, Denmark.

PMID: 24705213 PMCID: PMC3924827 DOI: 10.3390/genes4030457

Abstract

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 applicable method for gene-specific therapeutic regulation, and much effort has been put into further refinement of the technique. This review will focus on how RNAi has developed over the years and how the technique is exploited in a pre-clinical and clinical perspective in relation to neurodegenerative disorders.

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