Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue

Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue

The ability to deliver a therapeutic sequence to a specific cell type in the human brain would make possible innumerable therapeutic options for some of our most challenging diseases; however, studies on adeno-associated virus (AAV) vector tropism have generally relied on animal models with limited...

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Main Authors: JP McGinnis, Joshua Ortiz-Guzman, Maria Camila Guevara, Sai Mallannagari, Benjamin D.W. Belfort, Suyang Bao, Snigdha Srivastava, Maria Morkas, Emily Ji, Angela Addison, Evelyne K. Tantry, Sarah Chen, Ying Wang, Zihong Chen, Kalman A. Katlowitz, Jeffrey J. Lange, Melissa M. Blessing, Carrie A. Mohila, M. Cecilia Ljungberg, Guillermo Aldave, Ali Jalali, Akash Patel, Sameer A. Sheth, Howard L. Weiner, Shankar Gopinath, Ganesh Rao, Akdes Serin Harmanci, Daniel J. Curry, Benjamin R. Arenkiel
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
human brain
AAV
AAV tropism
human brain organotypic slice culture
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050125000890
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