Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment

Yang, X; Bui, TA; Mei, H; Aksoy, YA; Deng, F; Hutvagner, G; Deng, W

Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment

Yang, X Bui, TA Mei, H Aksoy, YA Deng, F Hutvagner, G

Deng, W

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Publisher:: WILEY-V C H VERLAG GMBH
Publication Type:: Journal Article
Citation:: Advanced Functional Materials, 2024
Issue Date:: 2024-01-01

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Field	Value	Language
dc.contributor.author	Yang, X
dc.contributor.author	Bui, TA
dc.contributor.author	Mei, H
dc.contributor.author	Aksoy, YA
dc.contributor.author	Deng, F
dc.contributor.author	Hutvagner, G https://orcid.org/0000-0002-7231-9446
dc.contributor.author	Deng, W https://orcid.org/0000-0002-9413-0978
dc.date.accessioned	2024-09-12T23:26:40Z
dc.date.available	2024-09-12T23:26:40Z
dc.date.issued	2024-01-01
dc.identifier.citation	Advanced Functional Materials, 2024
dc.identifier.issn	1616-301X
dc.identifier.issn	1616-3028
dc.identifier.uri	http://hdl.handle.net/10453/180803
dc.description.abstract	Human genetic disorders, arising from a range of genetic irregularities, can significantly affect human physiology, often with limited available treatment options. The development of the CRISPR system, facilitating precise editing of the genome, has opened new avenues for addressing a range of mutations found in various genetic disorders. However, there is currently a lack of comprehensive reviews that specifically address the application of CRISPR in genetic diseases. To bridge this gap, this review focuses on exploring the advancements in CRISPR technology and their utility in therapeutic approaches for various genetic disorders. This review introduces human genetic disorders, explains the fundamental mechanisms of CRISPR editing, and highlights the latest advancements in CRISPR technology. Additionally, it examines three CRISPR delivery techniques, including physical delivery, viral vectors, and nanocarriers. It further reviews CRISPR's applications in therapeutic approaches for genetic disorders. Finally, it identifies the primary hurdles associated with industrial development and ethics considerations that should be addressed before the application of CRISPR in a medical context.
dc.language	English
dc.publisher	WILEY-V C H VERLAG GMBH
dc.relation	http://purl.org/au-research/grants/nhmrc/1181889
dc.relation.ispartof	Advanced Functional Materials
dc.relation.isbasedon	10.1002/adfm.202402630
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	02 Physical Sciences, 03 Chemical Sciences, 09 Engineering
dc.subject.classification	Materials
dc.subject.classification	34 Chemical sciences
dc.subject.classification	40 Engineering
dc.subject.classification	51 Physical sciences
dc.title	Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment
dc.type	Journal Article
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/Strength - CHT - Health Technologies
pubs.organisational-group	University of Technology Sydney/Faculty of Engineering and Information Technology/School of Biomedical Engineering
pubs.organisational-group	University of Technology Sydney/All Manual Groups
pubs.organisational-group	University of Technology Sydney/All Manual Groups/Centre for Health Technologies (CHT)
utslib.copyright.status	open_access	*
dc.rights.license	This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0). To view a copy of this license, visit https://creativecommons.org/licenses/by-nc/4.0/
dc.date.updated	2024-09-12T23:26:39Z
pubs.publication-status	Published

Abstract:

Human genetic disorders, arising from a range of genetic irregularities, can significantly affect human physiology, often with limited available treatment options. The development of the CRISPR system, facilitating precise editing of the genome, has opened new avenues for addressing a range of mutations found in various genetic disorders. However, there is currently a lack of comprehensive reviews that specifically address the application of CRISPR in genetic diseases. To bridge this gap, this review focuses on exploring the advancements in CRISPR technology and their utility in therapeutic approaches for various genetic disorders. This review introduces human genetic disorders, explains the fundamental mechanisms of CRISPR editing, and highlights the latest advancements in CRISPR technology. Additionally, it examines three CRISPR delivery techniques, including physical delivery, viral vectors, and nanocarriers. It further reviews CRISPR's applications in therapeutic approaches for genetic disorders. Finally, it identifies the primary hurdles associated with industrial development and ethics considerations that should be addressed before the application of CRISPR in a medical context.

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http://hdl.handle.net/10453/180803