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US 2023/0414659 A1Published application
Methods of administering genetically modified b cells for in vivo delivery of therapeutic agents
OwnerChristiane S. HAMPEinventor
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Abstract
Provided herein are methods for administering engineered B cells to produce a therapeutic agent in vivo. In various embodiments, engineered B cells are directly administered to the central nervous system (CNS). The compositions and methods disclosed herein may be used for enzyme replacement therapy, for example, treatment of diseases or disorders associated with lysosomal storage dysfunction through production of iduronidase (IDUA).
Claims61
A method of administering genetically modified B cells to a subject for in vivo production of a therapeutic agent comprising:
- administering one or more doses of genetically modified B cells to a subject's central nervous system (CNS).
Dependencies are inferred from the claim text.