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

Liu, Y; Xia, X; Zheng, M; Shi, B

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

Liu, Y Xia, X Zheng, M Shi, B

Permalink

Publisher:: WILEY-V C H VERLAG GMBH
Publication Type:: Journal Article
Citation:: Adv Mater, 2024, 36, (29), pp. e2314354
Issue Date:: 2024-07

Closed Access

	Filename	Description	Size
	Advanced Materials - 2024 - Liu - Bio Nano Toolbox for Precision Alzheimer s Disease Gene Therapy.pdf	Published version	3.04 MB	Adobe PDF	View/Open

Copyright Clearance Process

Recently Added
In Progress
Closed Access

This item is closed access and not available.

Full metadata record

Field	Value	Language
dc.contributor.author	Liu, Y
dc.contributor.author	Xia, X
dc.contributor.author	Zheng, M
dc.contributor.author	Shi, B https://orcid.org/0000-0002-1043-3163
dc.date.accessioned	2024-11-05T04:32:59Z
dc.date.available	2024-11-05T04:32:59Z
dc.date.issued	2024-07
dc.identifier.citation	Adv Mater, 2024, 36, (29), pp. e2314354
dc.identifier.issn	0935-9648
dc.identifier.issn	1521-4095
dc.identifier.uri	http://hdl.handle.net/10453/181756
dc.description.abstract	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.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	WILEY-V C H VERLAG GMBH
dc.relation.ispartof	Adv Mater
dc.relation.isbasedon	10.1002/adma.202314354
dc.rights	info:eu-repo/semantics/closedAccess
dc.subject	02 Physical Sciences, 03 Chemical Sciences, 09 Engineering
dc.subject.classification	Nanoscience & Nanotechnology
dc.subject.classification	34 Chemical sciences
dc.subject.classification	40 Engineering
dc.subject.classification	51 Physical sciences
dc.subject.mesh	Alzheimer Disease
dc.subject.mesh	Humans
dc.subject.mesh	Genetic Therapy
dc.subject.mesh	Animals
dc.subject.mesh	Precision Medicine
dc.subject.mesh	Nanomedicine
dc.subject.mesh	Blood-Brain Barrier
dc.subject.mesh	Gene Transfer Techniques
dc.subject.mesh	Amyloid beta-Peptides
dc.subject.mesh	Nanoparticles
dc.subject.mesh	Blood-Brain Barrier
dc.subject.mesh	Animals
dc.subject.mesh	Humans
dc.subject.mesh	Alzheimer Disease
dc.subject.mesh	Gene Transfer Techniques
dc.subject.mesh	Nanomedicine
dc.subject.mesh	Nanoparticles
dc.subject.mesh	Amyloid beta-Peptides
dc.subject.mesh	Genetic Therapy
dc.subject.mesh	Precision Medicine
dc.subject.mesh	Alzheimer Disease
dc.subject.mesh	Humans
dc.subject.mesh	Genetic Therapy
dc.subject.mesh	Animals
dc.subject.mesh	Precision Medicine
dc.subject.mesh	Nanomedicine
dc.subject.mesh	Blood-Brain Barrier
dc.subject.mesh	Gene Transfer Techniques
dc.subject.mesh	Amyloid beta-Peptides
dc.subject.mesh	Nanoparticles
dc.title	Bio-Nano Toolbox for Precision Alzheimer's Disease Gene Therapy.
dc.type	Journal Article
utslib.citation.volume	36
utslib.location.activity	Germany
utslib.for	02 Physical Sciences
utslib.for	03 Chemical Sciences
utslib.for	09 Engineering
pubs.organisational-group	University of Technology Sydney
pubs.organisational-group	University of Technology Sydney/Faculty of Engineering and Information Technology
pubs.organisational-group	University of Technology Sydney/Faculty of Engineering and Information Technology/School of Biomedical Engineering
utslib.copyright.status	closed_access	*
dc.date.updated	2024-11-05T04:32:57Z
pubs.issue	29
pubs.publication-status	Published
pubs.volume	36
utslib.citation.issue	29

Abstract:

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.

Please use this identifier to cite or link to this item:

http://hdl.handle.net/10453/181756