Vectorology - GEG Tech top picks

Here, the scientists show that a somatic gene therapy using the gene for a modified U7 RNA which stimulates this splicing has a profound and persistent therapeutic effect on the phenotype of a severe Spinal Muscular Atrophy (SMA) mouse model. To this end, the U7 gene and AAV9 vector particles were designed. Introduction of the functional vector into motoneurons of newborn SMA mice by intracerebroventricular injection led to a highly significant, dose-dependent increase in life span and improvement of muscle functions. Besides the central nervous system, the therapeutic U7 RNA is expressed in the heart and liver which may additionally contribute to the observed therapeutic efficacy. This approach provides an additional therapeutic option for SMA and could also be adapted to treat other diseases of the central nervous systems with regulatory small RNA genes.