Osaka University

Nagoya Industrial Science Research Institute

Knc Laboratories Co., Ltd.

Aichi Medical University

Gen Sobue

Masahiro Neya

Kazuhiro Maeta

Tsuyoshi Fujihara

Seiji Matsuda

Yoshitaka Nagai

Toshihide Takeuchi

Shinsuke Ishigaki

Kentaro Sahashi

Masahisa Katsuno

Hiroaki Adachi

The present invention provides a suppression type antisense oligonucleotide targeting TDP-43 mRNA, containing a nucleotide sequence complementary to a sequence consisting of at least 10 continuous nucleotides in a nucleotide sequence shown in any of SEQ ID NOs: 2-4, and a promotion type antisense oligonucleotide targeting TDP-43 mRNA, containing a nucleotide sequence complementary to a sequence consisting of at least 10 continuous nucleotides in a nucleotide sequence shown in SEQ ID NO: 5.

Antisense oligonucleotide controlling expression amount of TDP-43 and use thereof

It is intended to provide a model animal faithfully reproducing the pathogenic conditions of spinal and bulbar muscular atrophy, a method of screening a remedy for polyglutamine disease using the same, and a remedy for spinal and bulbar muscular atrophy. Namely, a nonhuman animal having the following characteristics (1) to (5) in its conditions or pathological findings: (1) showing progressive myoatrophy; (2) showing lowering in muscular power; (3) in immunostaining with the use of an anti-polyglutamine antibody, showing nuclear diffuse staining and nuclear inclusions; (4) in immunostaining with the use of an anti-androgen receptor antibody, showing nuclear diffuse staining and nuclear inclusions; and (5) showing a neurogenic change. A remedy for polyglutamine disease is screened by administering a test substance to this nonhuman animal and examining changes in its conditions or pathological findings. A remedy for spinal and bulbar muscular atrophy is prepared by using as the active ingredient a compound having an effect of inhibiting the secretion of testosterone.

Methods for treating spinal and bulbar muscular atrophy using LHRH analogs

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Nagoya, Japan

Gen Sobue