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In vivo RNAi: Biodistribution, Delivery, and Applications
Allison O'Brien, PhD
RNA interference (RNAi) is an extremely valuable tool for the study of gene function, gene identification, and biological pathways. RNAi is an evolutionarily conserved antiviral response that is triggered by an externally introduced double stranded RNA (dsRNA). The dsRNA is cleaved by the RNase-III enzyme, Dicer, into small interfering RNAs (siRNA) that are approximately 21-23 nt. The siRNA then loads into an RNA-Induced Silencing Complex (RISC) which facilitates the separation of the two siRNA strands and cleavage of the target mRNA.
Synthetic siRNAs are utilized to suppress gene expression in mammalian cells without inducing an interferon response. siRNAs can be specifically designed to target any gene and can silence target mRNA expression often greater than 90%. The success of siRNA-mediated gene silencing in cell culture has led to the next application of RNAi, the use of synthetic siRNA for target-specific gene silencing in animals.
In vivo RNAi has been used for target validation studies in animal disease models. In vivo RNAi also has the potential to be used as siRNA therapeutics, where disease-causing genes could be selectively targeted and the gene expression suppressed. Diseases such as Alzheimers, cancer, age-related macular degeneration, retinopathy, asthma, chronic obstructive pulmonary disease, SARS, and rheumatoid arthritis would greatly benefit from siRNA therapeutics as the diseases are due to aberrant gene expression and the elimination of this gene expression could alleviate the disease.
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 In vivo RNAi (76K)
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