Gene Therapy vs. Gene Editing: Where is the Difference?

When talking genetics, gene therapy and gene editing are always mentioned and core. While both are groundbreaking for treating genetic disorders, they are fundamentally different in approach and applications. It is very crucial to understand their differences thus respective roles in genetics.

What is gene therapy? It can be defined as the treatment of disease by transfer of genetic material into cells. It involves introducing, removing, or altering genetic material within a person’s cells and aims to address the genetic cause of certain disorder. The goal is to replace the gene responsible for certain disorder with a health copy of the same usually achieved through a vector (viruses) which transfers the genetic materials into the patient’s cells.

What is gene editing? It involves altering specific DNA sequence of an organism’s genome or cell. Most common editing tool is the CRISPR-Cas9 that allows the target of specific gene. Gene editing is like a molecular “scissors” that allows researchers to precisely edit genetic material at specific locations in the genome. It can be more effective in correcting single-gene mutation related disorders such as Sickle cell anemia.

So, what are the key differences between gene therapy and gene editing?

Firstly, tools used. Gene editing or genome editing used tools like CRISPR-Cas9 to cut or edit DNA at desired or targeted locations allowing high degree of accuracy. Other editing tool includes TALENs. Gene therapy on the other hand uses vectors, mostly viruses to deliver genes into the cells. Nanoparticles may sometimes be used.

Secondly is the approach to the disease. Whereas gene editing directly modifies existing DNA sequence by making precise changes within the gene, usually allowing precise correction of mutations at the molecular level, gene therapy involves introducing a new, functional gene into a genome to replace a faulty one. It is particularly useful for treating recessive genetic disorders while gene editing is best for monogenic diseases.

Thirdly, mechanism of action. Gene therapy often go without altering the existing genome but rather focusing on delivering or adding new or modified genetic materials into the cell.  For example, to restore protein function in cystic fibrosis patient, a healthy copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is delivered into the patients’ cell. On the other hand, gene editing modifies the existing DNA sequence by making precise changes within the gene. For example, sickle cell anemia is corrected by editing a patient's own blood stem cells using CRISPR-Cas9, which specifically targets the gene responsible for producing abnormal hemoglobin, allowing the cells to produce more fetal hemoglobin which prevents red blood cells from sickling.

Other differences include permanency and ethical considerations.

gene therapy vs gene editing

In conclusion, although they are often mentioned together, each have distinctive approaches and are unique in their own way. Gene therapy offers a way to replace or add genes to treat genetic diseases, whereas gene editing can precisely alter the genome to correct mutations at the DNA level. Understanding these two technologies is crucial for researchers since they are continuously evolving.