The Role of RNAi in Target ID and Validation

RNA interference (RNAi) is an evolutionary regulatory mechanism in most cells, widely employed in high-throughput reverse genetics screening for basic research and drug discovery, and is also a powerful tool for target validation. RNAi screening methods have revolutionized the fields of basic biological research, functional genomics, and drug development and discovery. These methods provide complex and extremely powerful tools to study the underlying mechanisms of human eye disease and accelerate the development of treatments. In the field of drug discovery, RNAi impacts multiple stages of drug discovery, especially target identification and validation. It provides a method for the systematic study of gene families, signaling pathways, and biochemical pathways. RNAi screening can be used to identify novel drug targets and for target validation purposes. By using RNAi, it is possible to determine the function of a given gene in various cell culture systems. By knocking down a target mRNA with RNAi, it is feasible to understand whether the gene product encoded by that mRNA is involved in a pathway or process. In addition, it is possible to determine whether that gene or gene product is a suitable target for small-molecule intervention. Nowadays, state-of-the-art target validation experiments combine RNAi, DNA microarrays, and/or drug treatments to study the pleiotropic effects of knocking down a gene or combination of genes.

Fig. 1. Schematic illustration of arrayed or pooled RNAi screens in cells.(Seyhan, et al., 2020)

Services Overview

Ace Therapeutics has an RNAi technology team that focuses on drug target discovery to accelerate the discovery of targeted ophthalmological drugs. Our RNAi service design platform allows high levels of expression of shRNA and miRNA, which will help our customers conduct ophthalmology research, as follows:

• siRNA, shRNA and miRNA design and synthesis.
• The choice of mRNA and miRNA carriers.
• siRNA, shRNA and miRNA transfection optimization services.
• The custom preparation of shRNA and miRNA chronic virus particles and the establishment of stable cell lines.
• Include an experimental report that contains the RNAisequence, the results of the RT-PCR test, and the Western blot picture.

What Can We Offer?

• Libraries of various formats and types of RNAi agents for screening, including synthetic siRNAs directly transfected into cells as well as vector-encoded shRNAs.
• Multiple formats for RNAi library screening, including array screening as well as pooled screening.
• Genome-wide RNAi screens covering the entire human, mouse, and zebrafish genomes.
• Evaluation of specific and non-specific off-target effects.

Why Choose Us?

• Own subsets of gene families of interest, pharmacogenomics, or entire genomes of various organisms. RNAi panels can be tailored to target any number of genes, pathways, specific gene families (kinases, phosphatases, GPCRs, etc.), biological processes, or the entire genome of humans, mice, or rats.
• Comprehensive library screening landscape, including traditional transfection techniques and lentiviral vector transfection techniques.
• Provide array formats suitable for high-throughput readout.
• Pick genes of interest or screen entire libraries according to client project requirements.

Ace Therapeutics is committed to providing detailed data and comprehensive services for global customers' ophthalmic research. We are pleased to use our rich experience and advanced platforms to meet customers' every need. Please feel free to contact us if you have any special needs regarding target identification and validation for ophthalmic drug discovery. We look forward to working with you in the future.

Reference

1. Seyhan, Attila A. RNAi screens for the identification and validation of novel targets: Current status and challenges.Biology 2010;6.

• Computer-Aided Target Identification and Verification
• Gene Expression Profiling-Based Screening
• Genetic Engineering-Based Target Screening
• CRISPR/Cas9-Based Target Screening
• Label-Free Target ID
• Mechanism of Action (MOA) Study
• Target Functional Annotation and ID
• Target Structure Determination

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