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Updated: Jul 24, 2025

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
Oral non-viral gene delivery platforms for therapeutic applications
Asma Ghaemi1, Masoume Vakili-Azghandi2, Khalil Abnous3
1Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran; Department of Pharmaceutical Biotechnology, School of Pharmacy, Mashhad University of Medical Sciences, Mashhad, Iran.
Abstract:
Since gene therapy can regulate gene and protein expression directly, it has a great potential to prevent or treat a variety of genetic or acquired diseases through vaccines such as viral infections, cystic fibrosis, and cancer. Owing to their high efficacy, in vivo gene therapy trials are usually conducted intravenously, which is usually costly and invasive. There are several advantages to oral drug administration over intravenous injections, such as better patient compliance, ease of use, and lower cost. However, gene therapy is successful if the oligonucleotides can cross the cell membrane easily and reach the nucleus after the endosomal escape. In order to accomplish this task and deliver the cargo to the intended location, appropriate delivery systems should be introduced. This review summarizes oral delivery systems developed for effective gene delivery, vaccination, and treatment of various diseases. Studies have also shown that oral delivery approaches are potentially applicable to treat various diseases, especially inflammatory bowel disease, stomach, and colorectal cancers. Also, the current review provides an update overview on the development of non-viral and oral gene delivery techniques for gene therapy and vaccination purposes.
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