Base editing of ANGPTL3 and methods of treating disease using same

• Main Authors: RISE CAROLINE, RAJEEV KALLANTHOTTATHIL G, RODER ELLEN, CHADWICK ALEXANDRA, CHENG CHRISTOPHER
• Format: Patent
• Language: Chinese; English
• Published: 09.05.2023
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; SUGARS; VINEGAR; WINE

Provided herein are compositions for gene modification or editing and methods of using the same to treat or prevent certain conditions. Specific compositions and methods are disclosed that can safely and efficiently edit gene targets expressed in the liver to permanently reduce LDL-C, thereby treating the major cause of cardiovascular disease. 本文提供了用于基因修饰或编辑的组合物及其治疗或预防某些病症的使用方法。公开了能够安全且有效地编辑肝脏中表达的基因靶标以持久地降低LDL-C、从而治疗心血管疾病的主要病因的特定组合物和方法。