Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.

Autor: Ye D; Department of Biomedical Engineering, Washington University in St. Louis, Saint Louis, MO 63130, USA., Chukwu C; Department of Biomedical Engineering, Washington University in St. Louis, Saint Louis, MO 63130, USA., Yang Y; Department of Biomedical Engineering, Washington University in St. Louis, Saint Louis, MO 63130, USA., Hu Z; Department of Biomedical Engineering, Washington University in St. Louis, Saint Louis, MO 63130, USA., Chen H; Department of Biomedical Engineering, Washington University in St. Louis, Saint Louis, MO 63130, USA; Department of Neurosurgery, Washington University School of Medicine, Saint Louis, MO 63110 USA; Mallinckrodt Institute of Radiology, Washington University School of Medicine, Saint Louis, MO 63110, USA. Electronic address: hongchen@wustl.edu.
Jazyk: angličtina
Zdroj: Advanced drug delivery reviews [Adv Drug Deliv Rev] 2024 Aug; Vol. 211, pp. 115363. Date of Electronic Publication: 2024 Jun 19.
DOI: 10.1016/j.addr.2024.115363
Abstrakt: Adeno-associated virus (AAV) vectors have emerged as a promising tool in the development of gene therapies for various neurological diseases, including Alzheimer's disease and Parkinson's disease. However, the blood-brain barrier (BBB) poses a significant challenge to successfully delivering AAV vectors to the brain. Strategies that can overcome the BBB to improve the AAV delivery efficiency to the brain are essential to successful brain-targeted gene therapy. This review provides an overview of existing strategies employed for AAV delivery to the brain, including direct intraparenchymal injection, intra-cerebral spinal fluid injection, intranasal delivery, and intravenous injection of BBB-permeable AAVs. Focused ultrasound has emerged as a promising technology for the noninvasive and spatially targeted delivery of AAV administered by intravenous injection. This review also summarizes each strategy's current preclinical and clinical applications in treating neurological diseases. Moreover, this review includes a detailed discussion of the recent advances in the emerging focused ultrasound-mediated AAV delivery. Understanding the state-of-the-art of these gene delivery approaches is critical for future technology development to fulfill the great promise of AAV in neurological disease treatment.
Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.
(Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
Externí odkaz: