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New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery

The designer nucleases, including Zinc Finger Nuclease (ZFN), Transcription Activator-Like Effector Nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated (CRISPR/Cas), have been widely used for mechanistic studies, animal model generation, and gene therapy...

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Detalles Bibliográficos
Autores principales: Lyu, Pin, Lu, Baisong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9369418/
https://www.ncbi.nlm.nih.gov/pubmed/35955895
http://dx.doi.org/10.3390/ijms23158750
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author Lyu, Pin
Lu, Baisong
author_facet Lyu, Pin
Lu, Baisong
author_sort Lyu, Pin
collection PubMed
description The designer nucleases, including Zinc Finger Nuclease (ZFN), Transcription Activator-Like Effector Nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated (CRISPR/Cas), have been widely used for mechanistic studies, animal model generation, and gene therapy development. Clinical trials using designer nucleases to treat genetic diseases or cancers are showing promising results. Despite rapid progress, potential off-targets and host immune responses are challenges to be addressed for in vivo uses, especially in clinical applications. Short-term expression of the designer nucleases is necessary to reduce both risks. Currently, delivery methods enabling transient expression of designer nucleases are being pursued. Among these, virus-like particles as delivery vehicles for short-term designer nuclease expression have received much attention. This review will summarize recent developments in using virus-like particles (VLPs) for safe delivery of gene editing effectors to complement our last review on the same topic. First, we introduce some background information on how VLPs can be used for safe and efficient CRISPR/Cas9 delivery. Then, we summarize recently developed virus-like particles as genome editing vehicles. Finally, we discuss applications and future directions.
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spelling pubmed-93694182022-08-12 New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery Lyu, Pin Lu, Baisong Int J Mol Sci Review The designer nucleases, including Zinc Finger Nuclease (ZFN), Transcription Activator-Like Effector Nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated (CRISPR/Cas), have been widely used for mechanistic studies, animal model generation, and gene therapy development. Clinical trials using designer nucleases to treat genetic diseases or cancers are showing promising results. Despite rapid progress, potential off-targets and host immune responses are challenges to be addressed for in vivo uses, especially in clinical applications. Short-term expression of the designer nucleases is necessary to reduce both risks. Currently, delivery methods enabling transient expression of designer nucleases are being pursued. Among these, virus-like particles as delivery vehicles for short-term designer nuclease expression have received much attention. This review will summarize recent developments in using virus-like particles (VLPs) for safe delivery of gene editing effectors to complement our last review on the same topic. First, we introduce some background information on how VLPs can be used for safe and efficient CRISPR/Cas9 delivery. Then, we summarize recently developed virus-like particles as genome editing vehicles. Finally, we discuss applications and future directions. MDPI 2022-08-06 /pmc/articles/PMC9369418/ /pubmed/35955895 http://dx.doi.org/10.3390/ijms23158750 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Lyu, Pin
Lu, Baisong
New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title_full New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title_fullStr New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title_full_unstemmed New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title_short New Advances in Using Virus-like Particles and Related Technologies for Eukaryotic Genome Editing Delivery
title_sort new advances in using virus-like particles and related technologies for eukaryotic genome editing delivery
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9369418/
https://www.ncbi.nlm.nih.gov/pubmed/35955895
http://dx.doi.org/10.3390/ijms23158750
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