Gene therapy targeting the brain vasculature

Researchers have developed an engineered adeno-associated virus (AAV) vector that yields high transduction of brain vascular pericytes and smooth muscle cells. The study describing the characterization of this novel AAV capsid is published in the peer-reviewed journal Human Gene Therapy. Click here to read the article now.

In the current study, Servio Ramirez, from Temple University School of Medicine, Patricia Musolino, from Massachusetts General Hospital, and Casey Maguire, from Harvard Medical School, and coauthors, characterize AAV-PR, the capsid that demonstrated high transduction of the brain vasculature. AAV-PR offers the possibility of genetically modulating brain pericytes and smooth muscle cells in the context of neurodegeneration and other neurological diseases, according to the investigators. Many common neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, and amyotrophic lateral sclerosis, involve the brain vasculature.

“Because so many neurologic conditions stem from vascular dysfunction, the ability to deliver genes to the cells comprising these vessels could be truly paradigm shifting,” says 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 Chan 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 Chan 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

About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.