USE OF LENTIVECTOR-TRANSDUCED T-RAPA CELLS FOR AMELIORATION OF LYSOSOMAL STORAGE DISORDERS

• Main Authors: FOWLER DANIEL H, MEDIN JEFFREY A, NAGREE MURTAZA S, FELIZARDO TANIA
• Format: Patent
• Language: Chinese; English
• Published: 16.03.2021
• 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; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

The present disclosure provides methods of treating lysosomal storage disorders. The method comprises producing vector-transduced T-Rapa cells that express a transgene of interest and administering the cells to a patient in need thereof. 本公开提供了治疗溶酶体贮积症的方法。该方法包括产生表达目的转基因的载体转导的T-Rapa细胞，并将该细胞施用于有需要的患者。