MEDICINE FOR DISEASE CAUSED BY FRAME-SHIFT MUTATION

Abstract: the present disclosure provides a medicine for treating or preventing a hereditary disease, etc. the present disclosure provides a medicine for altering, preventing, or treating a condition, disease, disorder or symptom, which is caused by a frame-shift mutation due to the insertion or deletion of one or more bases, by cleaving at least one a target nucleic acid sequence in a nucleic acid within a cell, said medicine comprising a vector. this vector contains a construct that contains a promoter specific to the cell, a sequence encoding a cas nuclease, a promoter enabling the expression of a grna in the cell after the introduction of the vector, and a sequence encoding a grna that binds to a target nucleic acid sequence having the insertion or deletion. this construct is configured so as to form a cleavage site, by the cas nuclease, in proximity to the target nucleic acid sequence having the insertion or deletion.

Inventor(s): Koji Nishiguchi, Kosuke Fujita, Yuhei Ashida

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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