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Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. B...

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Autores principales: Dubey, Ankit Kumar, Mostafavi, Ebrahim
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10568484/
https://www.ncbi.nlm.nih.gov/pubmed/37841202
http://dx.doi.org/10.3389/fchem.2023.1259435
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author Dubey, Ankit Kumar
Mostafavi, Ebrahim
author_facet Dubey, Ankit Kumar
Mostafavi, Ebrahim
author_sort Dubey, Ankit Kumar
collection PubMed
description The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. Biomaterials play a vital role in shielding CRISPR/Cas9 components, such as lipid nanoparticles or viral vectors, from immunological processes and degradation, extending their effectiveness. By utilizing the flexibility of biomaterials, tailored systems can be designed to address specific genetic diseases, paving the way for personalized therapeutics. Furthermore, this delivery method offers promising avenues in combating viral illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate site-specific gene modifications, ensuring effective delivery to infected cells while minimizing off-target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target effects, ensuring long-term safety, and establishing scalable production techniques. Thorough research, pre-clinical investigations, and rigorous safety evaluations are imperative for successful translation from the laboratory to clinical applications. In this review, we discussed how CRISPR/Cas9 delivery using biomaterials revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities with the potential to significantly improve human health and quality of life.
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spelling pubmed-105684842023-10-13 Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy Dubey, Ankit Kumar Mostafavi, Ebrahim Front Chem Chemistry The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases holds tremendous potential. This innovative approach combines the advantages of CRISPR/Cas9 with the protective properties of biomaterials, enabling accurate and efficient gene editing while enhancing safety. Biomaterials play a vital role in shielding CRISPR/Cas9 components, such as lipid nanoparticles or viral vectors, from immunological processes and degradation, extending their effectiveness. By utilizing the flexibility of biomaterials, tailored systems can be designed to address specific genetic diseases, paving the way for personalized therapeutics. Furthermore, this delivery method offers promising avenues in combating viral illnesses by precisely modifying pathogen genomes, and reducing their pathogenicity. Biomaterials facilitate site-specific gene modifications, ensuring effective delivery to infected cells while minimizing off-target effects. However, challenges remain, including optimizing delivery efficiency, reducing off-target effects, ensuring long-term safety, and establishing scalable production techniques. Thorough research, pre-clinical investigations, and rigorous safety evaluations are imperative for successful translation from the laboratory to clinical applications. In this review, we discussed how CRISPR/Cas9 delivery using biomaterials revolutionizes gene therapy and infectious disease treatment, offering precise and safe editing capabilities with the potential to significantly improve human health and quality of life. Frontiers Media S.A. 2023-09-28 /pmc/articles/PMC10568484/ /pubmed/37841202 http://dx.doi.org/10.3389/fchem.2023.1259435 Text en Copyright © 2023 Dubey and Mostafavi. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Chemistry
Dubey, Ankit Kumar
Mostafavi, Ebrahim
Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title_full Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title_fullStr Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title_full_unstemmed Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title_short Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
title_sort biomaterials-mediated crispr/cas9 delivery: recent challenges and opportunities in gene therapy
topic Chemistry
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10568484/
https://www.ncbi.nlm.nih.gov/pubmed/37841202
http://dx.doi.org/10.3389/fchem.2023.1259435
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