Viruses are known to be dangerous pathogens which invade host cells and hi-jack their cellular machinery to direct the replication and transcription of their own genome. They are also known as being ideal couriers to transport new DNA into a host cell and ensure that it’s transcribed. Sophie Lutter at Oxgene looks into three aspects of viral biology that make them efficient delivery vehicles for gene therapies, and some of the ways that biologists have taken advantage of these properties to improve clinical performance.

Extract:

‘Viral Vector Engineering to Improve Clinical Performance and Accelerate Timeline to Success for Novel Gene Therapies’

Viruses are known to be dangerous pathogens which invade host cells and hi-jack their cellular machinery to direct the replication and transcription of their own genome. They are also known as being ideal couriers to transport new DNA into a host cell and ensure that it’s transcribed. Advanced therapy manufacturers have taken the head start that nature’s given them and used advances in genetic engineering, computational biology, and bioinformatics to perfect the design of the viral particle as a vector for therapeutic DNA and hasten the clinical success of ground-breaking new treatments. This article will look into three aspects of viral biology that make them efficient delivery vehicles for gene therapies, and some of the ways that biologists have taken advantage of these properties to improve clinical performance.

When efficient viral infection is a good thing

Maintaining open channels of communication between cells is essential for nutrient transport, signal transduction and other forms of cell-cell interaction, in normal physiological conditions. Viruses are adept at exploiting these channels to hitchhike into the cell. For example, a virus often used as a vector for gene replacement therapies such as adeno associated virus (AAV), enters the host cell by interacting with cell surface receptors, leading to endocytosis from clathrin coated pits. From there, a change in pH triggers the release of the virus from the endosome, where it is rapidly trafficked to the cell nucleus.

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