Title:A Review of Current and Prospective Treatments for Channelopathies, with a Focus on Gene and Protein Therapy
Volume: 29
Issue: 17
Author(s): Monica Sakla, Ulrike Breitinger, Hans-Georg Breitinger, Samar Mansour and Salma Nabil Tammam*
Affiliation:
- Department of Pharmaceutical Technology, The German University in Cairo (GUC), Cairo, Egypt
Keywords:
Channelopathies, nanomedicine, gene therapy, protein therapy, cellular machinery, symptomatic treatments.
Abstract: Reduced cell surface expression or the malfunctioning of ion channels gives rise to a group of disorders
known as channelopathies. To treat the underlying cause, the delivery and/or expression of a functional ion
channel into the cell membrane of the cell of interest is required. Unfortunately, for most channelopathies, current
treatment options are only symptomatic and treatments that rectify the underlying damage are still lacking.
Within this context, approaches that rely on gene and protein therapy are required. Gene therapy would allow
the expression of a functional protein, provided that the cellular machinery in the diseased cell could correctly
fold and traffic the protein to the cell membrane. Whereas protein therapy would allow the direct delivery of a
functional protein, provided that the purification process does not affect protein function and a suitable delivery
vehicle for targeted delivery is used. In this review, we provide an overview of channelopathies and available
symptomatic treatments. The current state of gene therapy approaches mainly using viral vectors is discussed,
which is followed by the role of nanomedicine in protein therapy and how nanomedicine could be exploited for
the delivery of functional ion channels to diseased cells.
