METHODS AND COMPOSITIONS FOR TREATING ANGELMAN SYNDROME

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 complementa...

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Bibliographic Details
Main Authors SIMON, JEREMY MARK, MAO, HANGQIAN, FRAGOLA, GIULIA, WOLTER, JUSTIN MATTHEW, ZYLKA, MARK JOHN
Format Patent
LanguageEnglish
French
Published 06.09.2019
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
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Summary: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. La présente invention concerne des méthodes et des compositions de traitement du syndrome d'Angelman, consistant à administrer à un sujet une quantité efficace d'une endonucléase associée à des répétitions palindromiques courtes, groupées et espacées régulièrement (CRISPR), ainsi qu'au moins une molécule d'ARN guide ayant une complémentarité avec une séquence nucléotidique cible dans des cellules UBE3A-ATS d'une sujet.
Bibliography:Application Number: CA20193091912