Ther Deliv. 2022 Sep;13(9):449-462. doi: 10.4155/tde-2022-0036. Epub 2023 Feb 7.
Wolfram syndrome is a rare multisystem autosomal recessive neurodegenerative disorder that affects the brain and central nervous system. Currently, there is no cure or treatment for Wolfram syndrome. Therefore, new techniques are needed to target the loss of the WFS1 gene. Gene therapy approach to introduce a functional gene using a viral or a non-viral vector could be a treatment strategy for Wolfram syndrome 1 (WS1). Viral vectors have therapeutic benefits and greater efficiency; however, they pose a high health risk. Recently pharmaceutical therapeutic research has developed cell-penetrating non-viral nano molecules that could be used as vectors for gene delivery. Among nonviral vectors, the unique properties of β-cyclodextrin suggest that it can be a promising safe vector for gene delivery.