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Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells
Advancements in gene delivery and editing have expanded the applications of autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and acquired diseases. The gene editing toolbox is growing, and the ability to achieve gene editing with mRNA or protein delivered int...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2023
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10540692/ https://www.ncbi.nlm.nih.gov/pubmed/37780116 http://dx.doi.org/10.3389/fgeed.2023.1148693 |
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author | Murugesan, Ramya Karuppusamy, Karthik V. Marepally, Srujan Thangavel, Saravanabhavan |
author_facet | Murugesan, Ramya Karuppusamy, Karthik V. Marepally, Srujan Thangavel, Saravanabhavan |
author_sort | Murugesan, Ramya |
collection | PubMed |
description | Advancements in gene delivery and editing have expanded the applications of autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and acquired diseases. The gene editing toolbox is growing, and the ability to achieve gene editing with mRNA or protein delivered intracellularly by vehicles, such as electroporation and nanoparticles, has highlighted the potential of gene editing in HSPCs. Ongoing phase I/II clinical trials with gene-edited HSPCs for β-hemoglobinopathies provide hope for treating monogenic diseases. The development of safe and efficient gene editing reagents and their delivery into hard-to-transfect HSPCs have been critical drivers in the rapid translation of HSPC gene editing into clinical studies. This review article summarizes the available payloads and delivery vehicles for gene editing HSPCs and their potential impact on therapeutic applications. |
format | Online Article Text |
id | pubmed-10540692 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-105406922023-09-30 Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells Murugesan, Ramya Karuppusamy, Karthik V. Marepally, Srujan Thangavel, Saravanabhavan Front Genome Ed Genome Editing Advancements in gene delivery and editing have expanded the applications of autologous hematopoietic stem and progenitor cells (HSPCs) for the treatment of monogenic and acquired diseases. The gene editing toolbox is growing, and the ability to achieve gene editing with mRNA or protein delivered intracellularly by vehicles, such as electroporation and nanoparticles, has highlighted the potential of gene editing in HSPCs. Ongoing phase I/II clinical trials with gene-edited HSPCs for β-hemoglobinopathies provide hope for treating monogenic diseases. The development of safe and efficient gene editing reagents and their delivery into hard-to-transfect HSPCs have been critical drivers in the rapid translation of HSPC gene editing into clinical studies. This review article summarizes the available payloads and delivery vehicles for gene editing HSPCs and their potential impact on therapeutic applications. Frontiers Media S.A. 2023-09-15 /pmc/articles/PMC10540692/ /pubmed/37780116 http://dx.doi.org/10.3389/fgeed.2023.1148693 Text en Copyright © 2023 Murugesan, Karuppusamy, Marepally and Thangavel. 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 | Genome Editing Murugesan, Ramya Karuppusamy, Karthik V. Marepally, Srujan Thangavel, Saravanabhavan Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title | Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title_full | Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title_fullStr | Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title_full_unstemmed | Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title_short | Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
title_sort | current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells |
topic | Genome Editing |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10540692/ https://www.ncbi.nlm.nih.gov/pubmed/37780116 http://dx.doi.org/10.3389/fgeed.2023.1148693 |
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