Engineered capsids for improved gene delivery to the retina and cornea

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.

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."

Terence R. Flotte, MD, Editor-in-Chief of Human Gene Therapy, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School

Journal reference:

Frederick, A., et al. (2020) Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea. Human Gene Therapy.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Breakthrough gene therapy offers hope for Duchenne muscular dystrophy