It is expected in the treatment of neurological disorders such as amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease, to be able to transfer as many therapeutic genes as possible to nerve cells in the brain and spinal cord. This rAAA makes gene transfer highly efficient by substituting the tyrosine residue at position 446 and 731 in coated protein of serotype 9 with a phenylalanine residue. By using a synapsin I promoter, rAAV makes gene expression occur in specific cells in the nervous system.
AAV is used in clinical research as a vector of gene therapy in the United States and Europe. It has attracted attention on a global basis because it is safe, non pathogenic and lacks the ability for viral growth. The Gene Therapy Research Institution is planning to treat neurological disorders such as ALS and Alzheimer’s disease at an early date by applying the patent “rAAV”, and lead the preceding gene therapy in the United States and Europe. The Gene Therapy Research Institution is expecting to perform this gene therapy not only in Japan but also overseas, especially Asia and the Middle East.
