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AAV therapy in muscle wasting conditions

Adeno-associated viruses (AAVs) are small viruses that infect humans and other primates but that are currently not known to cause any disease. 

About Adeno-associated viruses (AAVs)

They contain their own DNA and can inject it inside human cells. In fact, all viruses need to do this in order to ‘survive’. A virus is just made of genetic material packaged in a capsule. It needs to infect a host in order to reproduce. 

It is this feature of viruses that led scientists to think that they can be used as vectors, or carriers to deliver a new gene into cells. This approach is called gene therapy. In this approach, some of the viruses’ own DNA is replaced by the gene of interest. The virus carries the gene into the cell and injects it inside the cell. The cell then produces the protein encoded by the gene that the virus was carrying. AAVs can infect both dividing and non-dividing cells.

However, there are a few disadvantages in using AAVs for gene therapy. One of them is the small size of their genome, which is only 4,700 letters long. Therefore, they can only carry a small-sized DNA. Another disadvantage is the difficulty of producing them in large amounts in the laboratory. 

Even though AAVs are harmless and do not cause diseases, they are still a foreign material for the body and may cause a mild immune response especially if they are administered more than once. However, multiple administrations may not be necessary as the effect may be long lasting (many years) because the virus may persist in the body. The immune system may also attack the protein encoded by the gene that is being transferred because it has never come across it in the past. These problems may be overcome by drugs that suppress the immune response. 

To date, 12 naturally occurring human AAV strains and more than 100 non-human primates’ strains have been discovered. Different AAV strains have shown their preference in targeting different tissues. The availability of many strains increases the potential of AAVs as a delivery vehicle for gene therapy. 

Find out more about how drugs are developed and clinical trials. 

Clinical trials

Find out more about how drugs like AAV therapies are developed and clinical trials.

Genome editing

What genome editing is used for. The challenges, controversy and potential for treating muscle wasting conditions.