Antisense gene silencing: therapy for neurodegenerative disorders?

TY - JOUR

T1 - Antisense gene silencing

T2 - therapy for neurodegenerative disorders?

AU - Nielsen, Troels T

AU - Nielsen, Jørgen E

PY - 2013/9/10

Y1 - 2013/9/10

N2 - 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.

AB - 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.

U2 - 10.3390/genes4030457

DO - 10.3390/genes4030457

M3 - Journal article

C2 - 24705213

SN - 2073-4425

VL - 4

SP - 457

EP - 484

JO - Genes

JF - Genes

IS - 3

ER -