Technology Factsheet: Genome Editing

What is Genome Editing?

Genome Editing (also called “gene editing”) is defined as “a group of technologies that give scientists the ability to change an organism’s DNA.” These technologies edit DNA through adding, altering, or removing genetic material at target places inside the human genome. Genome editing is not new—scientists have used various techniques to create genetically modified organisms (GMOs) for decades. Yet the application to humans was limited because the methods were slow, costly, and not considered safe or reliable to deliver the treatment in humans. The discovery of a system called CRISPR-Cas9 led to faster, cheaper, and more accurate than existing gene editing systems such as ZFNs and TALENs. CRISPR-Cas9 was adapted from a naturally occurring genome editing system of bacteria used to protect cells from viral infections. CRISPR has been shown to work in human cells by cutting DNA at a predetermined target site, allowing scientists to then insert a different DNA sequence.

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