Israeli scientists use RNA-based gene therapy to stop ALS deterioration

By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers say the damaged nerve cells ‘even regenerated.’

The research was led by Dr. Ariel Ionescu, Dr. Lior Ankol, and lab manager Tal Pery Gradus in collaboration with Dr. Amir Dori, Senior Neurologist and Head of the Neuromuscular Disease Unit at Sheba Medical Center. Additional participants included researchers from the Weizmann Institute of Science, Ben-Gurion University of the Negev, and research institutions in France, Turkey, and Italy.

Their study, recently published in the peer-reviewed journal Nature Neuroscience, opens a new avenue for treating the disease.“We wanted to get to the root of the matter of what causes ALS to enable the development of effective drugs for this incurable disease,” Perlson said. The team used mice that had been genetically modified to serve as a “biological stand-in” for the disease. Read the complete article at the link above.