A research group led by Professor Norihiro Suzuki of the Department of Internal Medicine (Neuro), Keio University School of Medicine has succeeded in creating a new model mouse for amyotrophic lateral sclerosis (ALS).The therapeutic target was clarified by elucidating the onset mechanism.
ALS is an intractable disease in which motor nerves are impaired and the body cannot move, and there are about 9000 patients in Japan.About 90% are sporadic ALS with no relatives, and about 10% are familial (hereditary) ALS with relatives, the cause of the onset is unknown, and there is no radical cure.Research on impaired motor nerves is necessary to investigate the cause, but brain nerve cells are basically incapable of regeneration, and there is a limit to the analysis of motor nerves in prenatal ALS patients.Therefore, in order to elucidate the pathophysiology of ALS and develop treatment methods, it is important to analyze by producing mice that develop ALS (ALS model mice).
This time, the research group created mice (transgenic mice) into which FUS, which is one of the causative genes of familial ALS, was introduced.In mice, characteristic changes such as motor dysfunction occurring in actual ALS patients and reduction of motor nerves in brain tissue were observed.
Analysis of these mice demonstrated that the abnormally accumulated FUS in the cytoplasm exerted toxicity and directly damaged nerve cells.Until now, the involvement of FUS in the development of ALS and its exact mechanism have been unknown, but the true therapeutic target has been found to be abnormally accumulated FUS.Furthermore, analysis using mouse brain tissue revealed a group of genes whose expression levels were abnormally fluctuating.Since changes in this gene cluster are thought to be deeply involved in the onset of ALS, it is possible to use it as a marker for ALS diagnosis and develop therapeutic agents.
If the analysis of this model mouse progresses, it will be possible to develop a new treatment method for ALS and screen the efficacy and safety of the therapeutic drug, which will lead to the elucidation of the onset mechanism of ALS and the development of new diagnostic methods and new therapeutic drugs. Is expected.
