Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives
Monogenic disorders are a group of human diseases caused by mutations in single genes. While some disease-altering treatments offer relief and slow the progression of certain conditions, the majority of monogenic disorders still lack effective therapies. In recent years, gene therapy has appeared as...
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| Format: | Article |
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MDPI AG
2025-01-01
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| Series: | Pharmaceutics |
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| Online Access: | https://www.mdpi.com/1999-4923/17/1/104 |
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| author | Marco A. Luís Marcelo A. D. Goes Fátima Milhano Santos Joana Mesquita Paulo Tavares-Ratado Cândida Teixeira Tomaz |
| author_facet | Marco A. Luís Marcelo A. D. Goes Fátima Milhano Santos Joana Mesquita Paulo Tavares-Ratado Cândida Teixeira Tomaz |
| author_sort | Marco A. Luís |
| collection | DOAJ |
| description | Monogenic disorders are a group of human diseases caused by mutations in single genes. While some disease-altering treatments offer relief and slow the progression of certain conditions, the majority of monogenic disorders still lack effective therapies. In recent years, gene therapy has appeared as a promising approach for addressing genetic disorders. However, despite advancements in gene manipulation tools and delivery systems, several challenges remain unresolved, including inefficient delivery, lack of sustained expression, immunogenicity, toxicity, capacity limitations, genomic integration risks, and limited tissue specificity. This review provides an overview of the plasmid-based gene therapy techniques and delivery methods currently employed for monogenic diseases, highlighting the challenges they face and exploring potential strategies to overcome these barriers. |
| format | Article |
| id | doaj-art-24b9e6fe66ae46a08cfa85e30ed7416b |
| institution | Kabale University |
| issn | 1999-4923 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceutics |
| spelling | doaj-art-24b9e6fe66ae46a08cfa85e30ed7416b2025-01-24T13:45:58ZengMDPI AGPharmaceutics1999-49232025-01-0117110410.3390/pharmaceutics17010104Plasmid Gene Therapy for Monogenic Disorders: Challenges and PerspectivesMarco A. Luís0Marcelo A. D. Goes1Fátima Milhano Santos2Joana Mesquita3Paulo Tavares-Ratado4Cândida Teixeira Tomaz5CICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalCICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalCICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalCICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalCICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalCICS-UBI—Health Sciences Research Centre, University of Beira Interior, 6201-506 Covilhã, PortugalMonogenic disorders are a group of human diseases caused by mutations in single genes. While some disease-altering treatments offer relief and slow the progression of certain conditions, the majority of monogenic disorders still lack effective therapies. In recent years, gene therapy has appeared as a promising approach for addressing genetic disorders. However, despite advancements in gene manipulation tools and delivery systems, several challenges remain unresolved, including inefficient delivery, lack of sustained expression, immunogenicity, toxicity, capacity limitations, genomic integration risks, and limited tissue specificity. This review provides an overview of the plasmid-based gene therapy techniques and delivery methods currently employed for monogenic diseases, highlighting the challenges they face and exploring potential strategies to overcome these barriers.https://www.mdpi.com/1999-4923/17/1/104pDNAmonogenic diseasesnon-viral vectorgene therapy |
| spellingShingle | Marco A. Luís Marcelo A. D. Goes Fátima Milhano Santos Joana Mesquita Paulo Tavares-Ratado Cândida Teixeira Tomaz Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives Pharmaceutics pDNA monogenic diseases non-viral vector gene therapy |
| title | Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives |
| title_full | Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives |
| title_fullStr | Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives |
| title_full_unstemmed | Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives |
| title_short | Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives |
| title_sort | plasmid gene therapy for monogenic disorders challenges and perspectives |
| topic | pDNA monogenic diseases non-viral vector gene therapy |
| url | https://www.mdpi.com/1999-4923/17/1/104 |
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