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publicationDate 2021-02-25-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2021054370-A1
titleOfInvention Methods and compositions for treating angelman syndrome
abstract This invention relates to methods and compositions for treating Angelman syndrome, including administering to a subject an effective amount of a clustered regularly interspersed short palindromic repeat (CRISPR)-associated endonuclease and one or more than one guide RNA molecules having complementarity to a target nucleotide sequence in UBE3A-ATS in cells of the subject.
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