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METHODS FOR DETERMINING THE EFFICACY PROFILE OF A DRUG CANDIDATE
METHODS FOR DETERMINING THE EFFICACY PROFILE OF A DRUG CANDIDATE
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机译:确定药物候选药功效谱的方法
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摘要
The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
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