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filingDate 2019-12-27-04:00^^<http://www.w3.org/2001/XMLSchema#date>
grantDate 2022-01-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationDate 2022-01-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber CN-112996913-B
titleOfInvention Oligonucleotide molecules and their applications
abstract Small activating nucleic acid molecules and uses thereof for the treatment of spinal muscular atrophy are provided. The small activating nucleic acid molecule comprises a sense nucleic acid strand and an antisense nucleic acid strand, the sense nucleic acid strand and the antisense nucleic acid strand are oligonucleotide strands with a length of 16-35 nucleotides, respectively, and one of the nucleotide strands is connected to a selected oligonucleotide strand. There is at least 75% base homology or complementarity to the target site in the SMN2 promoter region of the target gene. Also provided is a pharmaceutical composition comprising a small activating nucleic acid molecule and optionally a pharmaceutically acceptable carrier; and using the small activating nucleic acid molecule or a pharmaceutical composition comprising the small activating nucleic acid molecule to upregulate the expression of a target gene in a cell and to treat the expression of the target gene Insufficient approach to disease caused by.
priorityDate 2018-12-29-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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