Gene editing will completely reshape molecular medicine within five years and it has already started.
The principle of gene editing is very simple: in the same way that spell check identifies and corrects errors in a word or grammar errors in a sentence, gene editing can be used to precisely edit or change the genetic code within an individual’s DNA.
Gene editing has many potential therapeutic applications. For example, it can be used to correct diseases and disorders that have a genetic basis. Just as editing text involves adding, removing, or replacing words, genome editing is an approach in which the genome sequence is directly changed by adding, replacing, or removing DNA bases. Gene editing is going to change the way people are treated by curing the roots of diseases instead of merely treating the symptoms. It provides us with the ability to rethink how we treat diseases altogether. It is the next transformative step in medicine.
There are three possible strategies to do this:
Insertion is used to add a new function to the genome. For example in drug discovery, or in order to overcome a genetic defect like hemophilia.
Correction is used to replace an existing defective sequence (which generally impacts the gene’s functions) by a functional sequence. For example, to treat a serious genetic disease such as cystic fibrosis.
Inactivation is used to prevent the expression of a gene. This approach can be used to treat persistent viral infections such as AIDS.