Chapters authored
First Insights into Non-invasive Administration Routes for Non-viral Gene Therapy
Gene delivery has attracted increasing interest as a highly promising therapeutic method to treat various diseases, including both genetic and acquired disorders. However, its clinical application is still hampered by the lack of safe and effective gene delivery techniques, as well as by the need of non-invasive routes of administration in gene delivery platforms. Among the different approaches used to transport nucleic acids into target cells, non-viral vectors represent promising and safer alternatives to viruses. Non-invasive administration routes are currently being studied, such as intranasal administration to target the brain, topical retinal administration for ocular diseases and aerosolized formulations for inhalation for the treatment of pulmonary diseases. Reasonable evidence suggests that future gene delivery systems might be based on effective non-viral vectors administered through non-invasive routes, which would constitute a safe, easy to produce, cheap and customizable alternative to the current viral gene delivery platforms. In this review, after briefly introducing the basis of gene therapy, we discuss the up-to-date and possible future strategies to improve DNA transfection efficiency using non-viral vectors and focusing on the non-invasive routes of administration.
Part of the book: Gene Therapy
Gene Therapy for Cystic Fibrosis: Hurdles to Overcome for Successful Clinical Translation
Cystic fibrosis (CF) is a genetic disease that hampers the lung function. Despite that the main defective gene has been deeply characterized, some relevant concerns still need to be resolved before considering gene therapy as a realistic medical choice. One of the major issues that need to be strongly considered in order to succeed in the search for an effective gene therapy approach for CF is the design of the appropriate genetic material to be delivered. Other relevant factors to take into consideration include the design of safe and effective gene delivery systems, the biological barriers that need to be overcome in order to reach the nucleus of the target cells, and the problems related to the design of a drug formulation suitable for lung delivery purposes. Furthermore, some problems related to the commercialization of gene therapy products also need to be resolved. In this chapter, we discuss the up-to-date strategies to overcome such hurdles in order for gene therapy to become a routine treatment modality for CF.
Part of the book: In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders