LentiVector-based gene therapy for Motor Neurone Disease
LentiVector-based gene therapy for Motor Neurone Disease - Project
Project overview
The research aims to determine whether MoNuDin is a viable treatment for sufferers of Motor Neurone Disease, for possible testing in future clinical trials. The preclinical stage has two specific objectives: a) to conduct laboratory studies in order to confirm safety and establish appropriate dosages for testing in patients; b) to establish a manufacturing process to generate sufficient MoNuDin for patient studies.