METHODS AND COMPOSITIONS FOR TREATING ANGELMAN SYNDROME

• Main Authors: FRAGOLA GIULIA, WOLTER JUSTIN MATTHEW, MAO HANGQIAN, ZYLKA MARK JOHN, SIMON JEREMY MARK
• Format: Patent
• Language: Chinese; English
• Published: 13.10.2020
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

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 repeats (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. 本发明涉及用于治疗Angelman综合征的方法和组合物，其包括向受试者施用有效量的成簇规律间隔短回文重复序列（CRISPR）相关的核酸内切酶以及一种或多于一种引导RNA分子，所述引导RNA分子与受试者的细胞中的UBE3A-ATS中的靶核苷酸序列具有互补性。