AB-302: Gene Therapy Vectors for Increased Genetic Payload and Long Term Expression

The effectiveness of a gene therapy is centered around the level and duration of expression of a therapeutic transgene. Viral vectors are used to deliver these transgene, and their expression is modulated by promoter sequences. Adenovirus-associated vectors are a commonly used viral vector for gene therapy due to their favorable immunogenicity profile, however, they are limited by their size of transgene able to be expressed, as well as expression levels. Prof. Engel at Princeton has developed a novel promoter that seeks to improve the AAV vector by show marked improvements in level and duration of gene expression than conventional promoter sequences.