Engineered adeno-associated virus vector yields high transduction of 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.

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."

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

Journal reference:

Ramirez, S. H., et al. (2023). An engineered AAV capsid mediates efficient transduction of pericytes and smooth muscle cells of the brain vasculature. 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
You might also like...
Evaluating the safety and efficacy of thalamic DBS in treating traumatic brain injury