A rational design strategy created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties within the mouse retina and cornea. as reported within the peer-reviewed journal Human Gene Remedy.
The environment friendly gene supply of those variants was confirmed in non-human primate tissue.
“The capsid modified AAV2 and AAV5 variants described right here have novel attributes that may add to the efficacy and specificity of their potential use in gene remedy for a spread of human ocular illnesses,” mentioned Catherine O’Riordan and coauthors from Sanofi.
The structural domains of the AAV capsid have grow to be elementary constructing blocks of many gene remedy vectors. The work by Dr. O’Riordan and her colleagues takes benefit of the brand new age of structural biology to intelligently redesign these constructing blocks slightly than counting on empiric screening of variants. This rational design strategy holds nice promise for the sector.”
Terence R. Flotte, MD, Editor-in-Chief of Human Gene Remedy, Celia and Isaac Haidak Professor of Medical Schooling and Dean, Provost, and Government Deputy Chancellor, College of Massachusetts Medical College
Frederick, A., et al. (2020) Engineered Capsids for Environment friendly Gene Supply to the Retina and Cornea. Human Gene Remedy. doi.org/10.1089/hum.2020.070.