AAV capsid-promoter interactions in the non-human primate brain

December 10, 2020

New Rochelle, NY, December 10, 2020--The phenomenon of AAV capsid-promoter interaction recently seen in the rat central nervous system has now been shown to occur in the non-human primate brain. This interaction can directly determine cell-specific transgene expression, as described in Human Gene Therapy. Click here to read the full-text article free on the Human Gene Therapy website through January 10, 2021.

An adeno-associated virus (AAV) contains a single-stranded DNA genome encapsulated in a capsid comprised of three structural proteins.

"We document a unique attribute of AAV vectors in both rodent and primate models that until recently remained undescribed: namely capsid/promoter interactions, that dictated cell type transduction profiles regardless of virus permissivity," state R. Jude Samulski, University of North Carolina School of Medicine, and coauthors.

"Up until now, we have thought of the AAV capsid as a 'delivery truck,' and once it dropped off its payload at the right cellular address, the vector promoter would do the rest. This work refutes that concept, showing that the capsid continues to have an effect on gene expression within specific cells of the brain and spinal cord. This has profound implications for vector design in the future," 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.

Research reported in this publication was supported by the National Institutes of Health under Award Numbers NEI R21, EY030278, EY013692, NS082289. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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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 newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Mary Ann Liebert, Inc./Genetic Engineering News

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