Developing a new gene therapy for periodic paralysis 

Muscle movement is caused when nerve cells send signals to the muscle. Periodic paralysis (PP) is a rare inherited condition in which these nerve signals fail to trigger the corresponding movement in muscle. As a result, people with PP experience recurring periods of muscle weakness and paralysis, the frequency of which increases with age. Although current treatments can limit how many times people experience muscle weakness/paralysis, they do not prevent the development of permanent muscle weakness later in life.  

The aim of this project is to develop a new gene therapy approach for PP. The researchers will test whether delivery of two different genes into muscle can reduce the development of temporary (episodic) and permanent muscle weakness in a mouse model of PP.  

If this project shows positive results in the mouse model, the researchers will look at taking it forward for further testing and future clinical trials in humans. This approach could have a beneficial impact for people living with PP as current treatments do not prevent the development of permanent muscle weakness and are not effective for everyone.