Gene therapy is an experimental technique that uses genes to treat or prevent disease. The most common form of gene therapy involves using DNA that encodes a functional gene to replace a mutated gene. Other forms involve directly correcting a mutation or encoding a therapeutic protein to provide a treatment. When this type of gene therapy is used, the therapeutic protein needs to be packaged within a “vector” which is used to transport the DNA inside cells within the body to treat a patients disease.


Ex Umbra In Solem | From The Shadow Into Light

Researchers at the University of New South Wales in Sydney, Australia are working to develop a vector using an Adeno Associated Virus (AAV) which is capable of transducing corrected genes with no immune response. The objective of the Joint Strike Vector Project is to develop a common delivery platform to specifically target oligodendrocytes, astrocytes and neurones in multiple tRNA-synthetase disorders. Vectors using an AAV can infect only dividing cells without integrating into the genome of the host cell, making them a very attractive candidate for gene therapies and for the creation of human disease models.