Plasmid Gene Therapy for Monogenic Disorders: Challenges and Perspectives

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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Bibliographic Details
Main Authors: Marco A. Luís, Marcelo A. D. Goes, Fátima Milhano Santos, Joana Mesquita, Paulo Tavares-Ratado, Cândida Teixeira Tomaz
Format: Article
Language:English
Published: MDPI AG 2025-01-01
Series:Pharmaceutics
Subjects:
pDNA
monogenic diseases
non-viral vector
gene therapy
Online Access:https://www.mdpi.com/1999-4923/17/1/104
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