Methods of genetically modifying cells for delivery of therapeutic proteins

• Main Authors: DUCHATEAU, PHILIPPE, JUILLERAT ALEXANDRE, BUSSER BRIAN, POIROT LAURENT, HUNG PATRICK, BOYEN ALEX
• Format: Patent
• Language: Chinese; English
• Published: 24.02.2023
• 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; SPIRITS; VINEGAR; WINE

The present disclosure provides methods of genetically modifying a cell by insertion of an artificial exon (ArtEx) for delivery of a therapeutic protein in a particular cell type, and more particularly, engineered cells for expression of a transgene to the brain of a patient. 本公开提供了通过插入人工外显子(ArtEx)对细胞进行基因修饰以便在特定细胞类型中递送治疗性蛋白质的方法，并且更特别地提供了用于将转基因表达到患者大脑的工程化细胞。