What is genome editing?

Genome editing uses a process which relies on the cell’s repair mechanism, i.e. the transformation of a DNA sequence prompts a living cell to achieve a desired outcome. This could be e.g. the correction of a malfunctional gene or the addition of missing cellular functions. The DNA transformation is achieved by a naturally occurring system that directs a biologic scissors, called a nuclease, to a specific region of the DNA. The nuclease cleaves the DNA. This triggers another natural process for every one of our cells which rebuilds the DNA. The cells’ repair machinery is there to repair any break that may occur within your DNA.

The DNA can be repaired in three different ways:

1) by inserting a DNA sequence, when a template DNA is provided to the cell in parallel with the targeted cleavage.

2) by deleting a DNA sequence, when two regions of DNA are cleaved;

3) by altering a DNA sequence;

The latest advances in genome editing have provided scientists with even more precise and efficient genome editing tools. These tools enable scientists to offer solutions to the numerous challenges we are facing in personal and public health, agriculture and the environment.

Medical scientists are exploring the potential of genome editing to treat and prevent life-threatening genetically-based diseases and conditions.