Intrathecal delivery of AAV-NDNF ameliorates disease progression of ALS mice

Amyotrophic lateral sclerosis (ALS) is a uniformly lethal neurodegenerative disease characterized by progressive deterioration of motor neurons, neuromuscular denervation, leading to severe paralysis and breathe failure. Adeno-associated virus (AAV)-mediated delivery of trophic factors is being considered as a potential disease-modifying therapeutic avenue. Here we show a marked effect of AAV-mediated over-expression of Neuron-derived neurotrophic factor (NDNF) on SOD1G93A ALS model mice. We performed gene expression profiling analysis using data obtained from RNA-seq of SOD1G93A mice injected with AAV-GFP or AAV-NDNF at postnatal days 74. Overall design: AAV-GFP or AAV-NDNF were intrathecally administrated into SOD1G93A mice at P74 and samples of the spinal cord ventral part were subjected to bulk RNA-sequence analysis at P104.