| Abstrakt: |
Simple Summary: Adenoviral vectors (AdVs) are engineered viruses used to deliver therapeutic genes to specific cells, offering promising solutions for the treatment of genetic diseases. However, their use is limited by issues such as strong immune responses and transient transgene expression. These limitations make them unsuitable for treatments that require long-term gene expression, such as some inherited diseases. This review examines clinical trials using AdVs for gene therapy of cystic fibrosis and ornithine transcarbamylase deficiency, their successes in preclinical testing and failures in practice, and discusses the underlying reasons for the failure of clinical trials. Understanding the reasons may help overcome these barriers to advances in gene therapy for inherited diseases. The review also highlights the achievements in overcoming these barriers. Scientists are modifying the outer structure of these vectors to more precisely target specific cells, attempting to reduce immune responses to AdVs, and improving gene delivery in cystic fibrosis by removing physical barriers such as thick mucus in the lungs. While these vectors are currently most useful for short-term applications such as vaccines and genome editing, ongoing research may open new doors for their use in more complex treatments. These advances have the potential to improve the effectiveness of gene therapy and offer hope to people living with incurable diseases. Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines. However, despite their numerous advantages, AdVs have not yet found their place in gene therapy for hereditary diseases. This review provides an overview of AdVs, their features, and clinical trials using them for gene replacement therapy in monogenic diseases and analyzes the reasons for the failures of these studies. Additionally, current research on the modification of AdVs to reduce immune responses and target delivery is discussed. [ABSTRACT FROM AUTHOR] |
