-A rational design approach created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties in the mouse retina and cornea. as reported in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free on the Human Gene Therapy website through September 13, 2020.
The efficient gene delivery of these variants was confirmed in non-human primate tissue.
“The capsid modified AAV2 and AAV5 variants described here have novel attributes that will add to the efficacy and specificity of their potential use in gene therapy for a range of human ocular diseases,” said Catherine O’Riordan and coauthors from Sanofi.
“The structural domains of the AAV capsid have become fundamental building blocks of many gene therapy vectors. The work by Dr. O’Riordan and her colleagues takes advantage of the new age of structural biology to intelligently redesign these building blocks rather than relying on empiric screening of variants. This rational design approach holds great promise for the field,” according to Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School.
About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.
About the Publisher
Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 90 journals, books, and news magazines is available on the Mary Ann Liebert, Inc., publishers website.