Overview
PackGene provides AAV vectors for reducing protein translation via short-hairpin RNA (shRNA), one of the most widely used RNAi methods inhibiting protein translation. We offer options to express shRNA under the H1 or U6 promoter, and all of our AAV-shRNA vectors are designed to co-express a reporter protein of your choosing.
RNA interference (RNAi) is a highly specific tool for protein knockdown in which via small non-coding RNA bind to target proteins mRNA to drive mRNA degradation and therefore reduce protein translation. RNAi mechanisms are endogenously present in most eukaryotes, and have been widely adopted as a molecular tool for gene function studies, drug discovery, and gene silencing therapy.
Several recent trials have shown that RNAi therapies that target disease-causing genes hold great potential for the treatment of human diseases. In comparison to other delivery methods, AAV vector-based expression of RNAi has been shown to drive prolonged reduction of mRNA targets in live animals.
PackGene can assist customers with shRNA vector design and vector construction. Please inquire for more details.
Direct, convenient, and efficient expression of shRNA for in vivo animal experiments
Ability to target various organs and tissues through specific AAV serotypes
Geater expression durability and longer effect duration when compared to traditional synthetic shRNA +
Increased safety and immunogenicity relative to traditional synthetic shRNA
PackGene offers a guarantee that our triple shRNA expression AAVs will reduce target mRNA expression by 70% or more in cases where target-cell transfection efficiency is 90% or greater*.
In a case where our AAV-shRNA guarantee is not met, PackGene will design two new interfering sequences for you free of charge**.
* Protein expression level is not guaranteed. Disruptions in the efficacy of non-coding genes is not guaranteed.
** Customer must provide clear data demonstrating inefficiency of mRNA knockdown.
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