Bio-Nano Toolbox for Precision Alzheimer's Disease Gene Therapy.

Autor: Liu Y; Department of Radiotherapy and Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, Henan, 475000, China.; Henan-Macquarie University Joint Centre for Biomedical Innovation, School of Life Sciences, Henan University, Kaifeng, Henan, 475004, China., Xia X; Department of Radiotherapy and Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, Henan, 475000, China.; Henan-Macquarie University Joint Centre for Biomedical Innovation, School of Life Sciences, Henan University, Kaifeng, Henan, 475004, China.; Macquarie Medical School, Faculty of Medicine & Health Sciences, Macquarie University, Sydney, New South Wales, 2109, Australia., Zheng M; Department of Radiotherapy and Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, Henan, 475000, China.; Henan-Macquarie University Joint Centre for Biomedical Innovation, School of Life Sciences, Henan University, Kaifeng, Henan, 475004, China., Shi B; Henan-Macquarie University Joint Centre for Biomedical Innovation, School of Life Sciences, Henan University, Kaifeng, Henan, 475004, China.; Macquarie Medical School, Faculty of Medicine & Health Sciences, Macquarie University, Sydney, New South Wales, 2109, Australia.
Jazyk: angličtina
Zdroj: Advanced materials (Deerfield Beach, Fla.) [Adv Mater] 2024 Jul; Vol. 36 (29), pp. e2314354. Date of Electronic Publication: 2024 May 22.
DOI: 10.1002/adma.202314354
Abstrakt: Alzheimer's disease (AD) is the most burdensome aging-associated neurodegenerative disorder, and its treatment encounters numerous failures during drug development. Although there are newly approved in-market β-amyloid targeting antibody solutions, pathological heterogeneity among patient populations still challenges the treatment outcome. Emerging advances in gene therapies offer opportunities for more precise personalized medicine; while, major obstacles including the pathological heterogeneity among patient populations, the puzzled mechanism for druggable target development, and the precision delivery of functional therapeutic elements across the blood-brain barrier remain and limit the use of gene therapy for central neuronal diseases. Aiming for "precision delivery" challenges, nanomedicine provides versatile platforms that may overcome the targeted delivery challenges for AD gene therapy. In this perspective, to picture a toolbox for AD gene therapy strategy development, the most recent advances from benchtop to clinics are highlighted, possibly available gene therapy targets, tools, and delivery platforms are outlined, their challenges as well as rational design elements are addressed, and perspectives in this promising research field are discussed.
(© 2024 Wiley‐VCH GmbH.)
Databáze: MEDLINE
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