Gene-therapy approaches

What is gene editing?

Gene editing is a type of gene therapy. This technique aims to change the body’s genetic material at specific locations in the DNA. The goal of gene editing is to modify gene mutations inside the body’s cells.

Modifying a gene

Gene editing occurs when scientists make small changes in DNA. Earlier, we discussed how your genes are like an instruction manual. Imagine reading a page in that manual and coming across an incorrect word.

To fix it, you change the faulty word within the sentence. For example, you may need to cross out the word (turning it off), change it, or add a word, so the sentence makes sense.

Once you make the needed change, the instruction manual is edited. Gene editing works similarly. The goal is to edit genes to manage or treat a genetic disease.

Instruction manual illustration

How gene editing works

ZFNs illustration next to DNA strand

Target

A protein is programmed to find a specific part of DNA. It scans the DNA until it finds the specific site.

DNA strand broken at the targeted location

Break

The protein creates a break at the targeted location. This allows the gene-editing mechanism to work.

A pencil targeting a specific strand of DNA representing gene correction

Correct

Gene editing technologies may correct the mutation by turning a gene on or off. They may also provide a new section of DNA to repair a mutation.

Protein being produced by a DNA strand

Build

The newly edited gene tells the body how to build proteins to potentially change the way the body functions.

There are several methods of gene editing being studied. These include:

  • ZFNs (zinc finger nucleases)
  • TALENs (transcription activator-like effector nucleases)
  • CRISPR (clustered regularly interspaced short palindromic repeats)

To learn more about these methods, visit the Learning Center.

With this overview of gene editing in mind, it’s time to take a closer look at 1 type of gene editing.

Explore the CRISPR/Cas system

Learn more

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