Next-generation muscle-directed gene therapy by in silico vector design

Next-generation muscle-directed gene therapy by in silico vector design

Next-generation muscle-directed gene therapy by in silico vector design, Published online: 30 January 2019; doi:10.1038/s41467-018-08283-7

Adeno-associated viral vectors (AAV) are being developed for gene therapy of skeletal muscle, but it is a challenge to achieve robust gene expression. Here, the authors identify muscle-specific cisregulatory elements that lead to a substantial increase in micro-dystrophin and follistatin expression, resulting in a safe and sustainable rescue of the dystrophic phenotype in mouse models.

from A via a.sfakia on Inoreader https://go.nature.com/2MI5Ofn