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Engineered capsids for efficient gene delivery to the eye

 


-A rational design approach has created a new variant of the adeno-associated virus (AAV) capsid. They improve the transduction properties of the mouse retina and cornea. As reported in the peer-reviewed journal Human Gene Therapy. click here Read the full article, free of charge, on the Human Gene Therapy website until September 13, 2020.

Efficient gene delivery of these mutants was confirmed in non-human primate tissues.

“The capsid-modified AAV2 and AAV5 variants described here have new attributes that add to the efficacy and specificity of their potential use in gene therapy for various human eye diseases,” Catherine O ‘Co-authors of Riordan and Sanofi said.

“The structural domain of the AAV capsid has become a basic building block in many gene therapy vectors. The work of Dr. O’Riordan and his colleagues has taken advantage of a new era in structural biology to study empirical variants. Instead of relying on screening, we intelligently redesign these building blocks. This rational design approach holds great promise in this area,” the editor-in-chief said. Human gene therapy Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education, Dean, Vice-President of the University of Massachusetts School of Medicine.

About the journal

Human gene therapy, The official journal of the European Society for Gene and Cell Therapy and eight other international gene therapy societies are the first peer-reviewed journals in this field and offer comprehensive access to the key pillars of human gene therapy. Masu: research, methods, and clinical applications. The journal is headed by Dr. Terence R. Float, Professor of Medical Education, Celia Isaac Heidak, Professor, Vice President of Medicine, University of Massachusetts, and an international editorial board. Human gene therapy Available in print and online. The complete table of contents and sample questions are Human gene therapy website.

About publishers

Mary Ann Liebert, Inc., Publisher Known for establishing prestigious peer-reviewed journals in many promising areas of science and biomedical research. The biotechnology industry magazine, GEN (Genetic engineering and biotechnology news) Is the first in its field and is today the most widely read publication in the industry. For a complete list of the company’s 90 journals, books and news magazines, Mary Ann Liebert, Inc., Publisher website.

/ Public release. The material in this public release is from the original organization, is at a specific point in time and may be edited for clarity, style, or length. View full text here..

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