R&D Program Overview

Research&Development R&D Program Overview

Engineered Nuclease

R&D Program Overview




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ToolGen has been doing business based on genome editing technology. ToolGen has been shaping itself as the specialized company which has succeeded in developing 1st, 2nd and 3rd generation genome editing technology progressively.

Engineered Nucleases

We believe that the most powerful molecular tools enabling targeted cleavage in genome for efficient genome editing are engineered nucleases. For the last decade, engineered nuclease technologies have been developed exponentially to enable us to edit genome efficiently. There are two essential things in engineered nuclease-mediated genome editing. First thing is targeting specific sequence among billions of DNA bases precisely and second thing is cleavage occurring at targeted sites. Through targeted cleavage, it enables gene knockout, gene correction, gene modification and gene addition at intended location.

CRISPR/Cas9 Technology

Since the discovery of CRISPR/Cas9, the 3rd generation of engineered nuclease, CRISPR/Cas9 has rapidly expanded into various scientific area with extensive increase of interest on its improvement from previous generations and it leads us to much simpler and more precise genome editing methodology. CRISPR/Cas9 is novel, programmable nuclease platform revolutionizing the genome editing technology.
· Key Features
· Structure & Mechanism of CRISPR/Cas9
Adapted from bacterial immune machinery, we developed a CRISPR/Cas9 nuclease platform which consist of a target-specific RNA (guide RNA) and Cas9 protein. In this CRISPR/Cas9 system, single guide RNA plays a key role in finding targeted site where cleavage should be occurred.
· Application
And then Cas9 proteins cut the targeted DNA sites by their nuclease domains. Through targeted cleavage by CRISPR/Cas9 system, it enables gene knockout, gene correction, gene modification and gene addition at intended location. By customizing guide RNA, CRISPR/Cas9 can be programmed to bind and cut a specific site of genome enabling efficient editing of genetic information.

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