MAAsiRNA

A significant challenge in the field of nervous system pathologies lies in the absence of effective treatments. MAAsiRNA develops a pioneering targeted therapy utilizing small interfering RNA (siRNA). After over a decade of intensive interdisciplinary research, we have successfully developed a siRNA-based treatment for Charcot-Marie-Tooth disease type 1A (CMT1A), the most prevalent form of hereditary peripheral neuropathy. CMT1A affects 1 in 5000 people, comprising a total of 1.5 million individuals worldwide. Our current objective is to advance towards a patient-ready therapy. Our innovative therapeutic approach has the potential to be adapted for other disorders within the nervous system.