The Modification of AAV’s for Safer Gene Therapy Treatments

We are brought up believing that all viruses and bacteria are harmful. This is at least until we learn that various vaccines administered to us over our lifetimes are done so using viral vectors. The use of viruses as modes of delivery is mainly because of their genetic makeup. This feature is also what makes them useful for gene therapy.

A study carried out by James Wilson, a professor of medicine and April R Giles, who also happen to be the senior and first authors, respectively, on a paper on the same, has led to some exciting discoveries. It has been noted that during the harvesting and preparation of viral vectors, during the manufacturing processes, these organisms lose some of their activity.

Currently, the most common viral vector for gene therapy is the adeno-associated virus. This is made up of 60 interlocking proteins, which form a capsid around the gene being delivered.  These proteins will then allow for the attachment and entry into the targeted cells and the delivery of the gene. The current problem is the spontaneous mutation of these proteins, which can affect the delivery of the gene.

This study also uncovered that the outer AAV’s proteins, which were long assumed to be the same are different. To better understand this, the research team used traditional methods to determine the specific biochemical nature of these proteins and the virus as a whole. In addition to this, they are also working on developing capsid proteins that are not susceptible to spontaneous mutation.

The development of these capsid proteins is something many in the field of genetic will applaud. This is because apart from raising the chances of safe gene therapy treatments, it will also cut the financial costs of these therapies and minimize the adverse side effects.

Reference

https://www.sciencedaily.com/releases/2018/10/181018141038.htm

 

 

 

 

 

 

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