The invention discloses an inhibitor acting on a TDP1 target spot and application of the inhibitor. The
active ingredient of the inhibitor comprises Lawsone (2-hydroxy-1, 4-
naphthoquinone) or SPI-112 (
Sodium Sulfonate I-112). The Lawsone is a
naphthoquinone compound with oral activity, has antibacterial, antitumor and
antioxidant activity, has a molecular formula of C10H6O3, and has a molecular weight of 174.16. The SPI-112 is a
protein tyrosine phosphatase inhibitor, the molecular formula of the SPI-112 is C22H17FN4O5S, and the molecular weight of the SPI-112 is 468.46. The inhibitor provided by the invention can be used for effectively reducing CTG trinucleotide repeated amplification in Mykylosing Dystrophy Type 1 (Mykylosing Dystrophy Type 1, DM1) by inhibiting the activity of TDP1
protein, and a new direction is provided for preparing a safe and effective
medicine directly aiming at the CTG trinucleotide repeated amplification in DM1.