Genome Editing:CRISPR

Technology to modify a gene by cutting it at a target site

Genome editing is a technology that modifies a gene by cutting the double strand of DNA at a specific point and either 1) destroying the target gene or 2) repairing it by inserting a different sequence at the point of breakage.

Molecules can be designed for any cleavage site by simply replacing the gRNA for each target.

The CRISPR/Cas genome editing system consists of two components: a cleavage enzyme called Cas and a guide RNA (gRNA), which is a positioning nucleic acid.

By designing a gRNA that matches the sequence of the desired cleavage site on the DNA, a complex of Cas and gRNA binds to the target site and performs the cleavage.

Although there were genome editing technologies such as ZFN and TALEN before CRISPR/Cas, the ability to perform pinpoint editing anywhere by simply replacing the gRNA makes CRISPR/Cas much more efficient than conventional methods such as ZFN and TALEN in terms of simplicity of design, improved throughput, multiple editing, and high efficiency. The technology is much improved in terms of design simplicity, throughput, multiple editing, high efficiency, and a wide range of applications compared to conventional methods such as ZFN and TALEN.

There are other CRISPR systems besides Cas9, such as Cas3, but they are not suitable for gene therapy due to their low accuracy, extensive degradation, and other issues.

MODALIS aims to use the best system and to treat patients in the best possible way.