According to a new scientific publication in Cell, a team of researchers have engineered a new gene-shuttling virus, known as MyoAAV, which has the potential to deliver gene therapy more safely in humans, by targeting muscle tissue at lower doses – between 100-250 times lower than current approaches. The research team, led by Dr Sharif Tabebordbar are based at Broad Institute of MIT and Harvard, and Harvard University in US. You can read the full publication here: Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species.
A more simple article, explaining how this new approach using MyoAAV works, together with a video about how Dr Tabebordbar was inspired to work in this field, can be found here on the research team’s own academic website: https://www.broadinstitute.org/news/new-gene-delivery-vehicle-could-make-gene-therapy-muscle-diseases-safer-and-more-effective
This is particularly hopeful and welcome news in light of recent tragic events in the AT132 Gene Therapy trials (ASPIRO), when it was announced that a 4th child sadly died following dose using the vector AAV8.
Photo credit: Broad Institute’s Communications Team
