Gene therapy

What is gene therapy?

Gene therapy is a method of treating the genes that cause diseases at the normal gene level, and is expected to be the fundamental treatment for cancer and congenital diseases.

Methods of gene therapy

So, how is gene therapy actually done? The first step in gene therapy is to analyze the genes and diagnose the possibility of diseases. If a disease-causing gene is found, a gene suitable for the patient is introduced into the cells in order to suppress the disease-causing gene or normalize the gene.

How to introduce the gene

Now that we have explained the general flow of gene therapy, how exactly do we introduce genes into cells? One of the main methods is the use of viral vectors. In the method using viral vectors, the gene to be introduced is embedded in a virus, and the virus infects the target cell to introduce the gene.

Problems with viral vectors

However, there are problems with viral vectors. There are several types of viral vectors, including adeno-associated virus (AAV), adenovirus, retrovirus, and lentivirus, but each has its own drawbacks. Please see the table below.

Viruses	Advantages	Disadvantages
Retrovirus	・Gene expression lasts forever	・Unstable and may become cancerous ・Cannot be used for non-dividing cells
Adenoviruses	・High expression efficiency ・Can be used in non-dividing cells	・Long-lasting gene expression
Lentivirus	・Long-lasting gene expression ・Can be used for non-dividing cells	・Uses HIV virus ・Complicated production method
Adeno-associated virus	・Long-lasting gene expression ・Can be used for non-dividing cells	・Small number of genes that can be inserted

As you can see from the table, each virus has its own advantages and disadvantages. These viruses need to be selected according to their intended use.

Viruses, which are usually our enemies, have been used for gene therapy. However, it is far from widespread and new gene therapies need to be developed.

	What will be the future of gene therapy?