Ractigen Therapeutics (20250101421). OLIGONUCLEOTIDE MODULATORS ACTIVATING UTROPHIN EXPRESSION
OLIGONUCLEOTIDE MODULATORS ACTIVATING UTROPHIN EXPRESSION
Organization Name
Inventor(s)
Moorim Kang of Nantong Jiangsu CN
Robert Place of Nantong Jiangsu CN
Longcheng Li of Nantong Jiangsu CN
OLIGONUCLEOTIDE MODULATORS ACTIVATING UTROPHIN EXPRESSION
This abstract first appeared for US patent application 20250101421 titled 'OLIGONUCLEOTIDE MODULATORS ACTIVATING UTROPHIN EXPRESSION
Original Abstract Submitted
provided is an oligonucleotide modulator for preventing or treating dystrophin-deficient-related disorders (ddd) including duchenne muscular dystrophy (dmd) and becker muscular dystrophy (bmd). the oligonucleotide modulator comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid strand and the antisense nucleic acid strand are independently an oligonucleotide strand of 16 to 35 nucleotides in length, in which one nucleotide strand has at least 75% base homology or complementarity to a target selected from a promoter region of a target gene utrn.
