PROGRAMMABLE RNA EDITING IN VIVO VIA RECRUITMENT OF ENDOGENOUS ADARS

• Main Authors: MALI, Prashant, YEN, James, KATREKAR, Dhruva
• Format: Patent
• Language: English; French
• Published: 07.07.2022
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS OR TEST PAPERS THEREFOR; COMPOSITIONS THEREOF; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROCESSES OF PREPARING SUCH COMPOSITIONS; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Disclosed herein are engineered guide RNAs, constructs for forming engineered guide RNAs, pharmaceutical compositions thereof, methods of making the engineered guide RNAs, and methods of treating or preventing a diseases and disorders of a subject by administering one or more of the engineered guide RNAs or the constructs for forming the engineered guide RNAs. Sont divulgués ici des ARN guides modifiés, des constructions pour former des ARN guides modifiés, des compositions pharmaceutiques associées, des procédés de fabrication des ARN guides modifiés, et des méthodes de traitement ou de prévention de maladies et de troubles chez un sujet par l'administration d'un ou plusieurs des ARN guides modifiés ou des constructions pour former les ARN guides modifiés.