# What is the difference between gene therapy and genome editing?

Canonical page: https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing
Language: en
Category: general
Publisher: ClinicsJo
Published: 2026-09-13T17:52:53.585Z
Content updated: 2026-09-13T17:52:53.585Z
Translation (ar): https://clinicsjo.com/ar/faq/what-is-the-difference-between-gene-therapy-and-genome-editing

Gene therapy and genome editing are two approaches that aim to treat genetic diseases by altering genetic material, but they differ in their method of action. Traditional gene therapy involves introducing new genes into cells to compensate for defective or missing genes, or to add a new function to the cells. In contrast, genome editing focuses on precisely altering the DNA already present within the cells, allowing for the addition, removal, or modification of specific parts of the genome [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>).

Both approaches aim to correct genetic problems that cause diseases, such as replacing an abnormal gene with a normal one, or modifying the activity level of a specific gene. However, genome editing, such as the CRISPR-Cas9 technique, represents a newer development that allows for more precise and targeted modifications within the cell's original DNA [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>).

## How does gene therapy work?

Gene therapy works by introducing new genetic material into cells. This can be done in two main ways: ex-vivo therapy, where cells are removed from the body, modified by adding genetic material, and then returned to the body; or in-vivo therapy, where the genetic material is delivered directly into the body, often via injection [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). Modified viruses are often used as vectors to deliver this genetic material to target cells, after removing their ability to cause disease [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>). Gene therapy can compensate for disease-causing genetic changes by introducing a normal version of the gene to restore the function of a necessary protein, or by introducing a different gene that provides instructions for a protein that helps the cell function normally [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>).

## How does genome editing differ?

Genome editing is a newer and more specific technique that does not necessarily add entirely new genetic material; instead, it introduces gene-editing tools that can change the DNA already present in the cell [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>). Genome editing techniques allow for the addition, removal, or alteration of genetic material at precise and specific locations within the genome [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>). The CRISPR-Cas9 technique is a well-known example of genome editing and is promising for the treatment of genetic disorders [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). These tools can correct errors in DNA and restore protein function [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>).

## Challenges and Risks

Despite the great promise of both gene therapy and genome editing, they face challenges and risks. The body's immune system may react to the new genetic material or viral vectors, perceiving them as a threat [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). Changes in DNA could lead to unintended effects on other genes in harmful ways [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). Furthermore, gene therapies are often expensive and not widely available [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). Researchers are constantly working on developing better and more targeted delivery techniques, such as the use of nanoparticles, to reduce risks and increase efficacy [\[2\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S2>).

## The Future of Gene Therapy and Genome Editing

Both gene therapy and genome editing are rapidly evolving fields that hold immense potential for treating and preventing genetic diseases [\[3\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S3>). Currently, gene therapies have been approved for treating a small number of diseases, such as [spinal muscular atrophy](<https://clinicsjo.com/ar/articles/gene-therapy-future-genetic-diseases>) (Arabic) and some hereditary eye disorders [\[1\]](<https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#source-S1>). As scientific and technological progress continues, the list of diseases that can be treated with these techniques is expected to expand. It is important to consult specialist physicians to discuss available treatment options and assess their suitability for an individual case, as these treatments require careful clinical monitoring.

## Medical references

- [Genes and Gene Therapy](<https://medlineplus.gov/genesandgenetherapy.html>)

- [How does gene therapy work?: MedlinePlus Genetics](<https://medlineplus.gov/genetics/understanding/therapy/procedures/>)

- [How Gene Therapy Can Cure or Treat Diseases](<https://www.fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases>)

General health education; not a substitute for individualized clinical assessment.

Citation URL: https://clinicsjo.com/en/faq/what-is-the-difference-between-gene-therapy-and-genome-editing#answer