Adjustable gene therapy
AssigneeRemedium Bio, Inc.
InventorsAlexei Goraltchouk, Alexey Seregin, Frank Luppino
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Abstract
The present invention applies, at least in part, to therapeutic agents, and more specifically to gene therapy for use in human or veterinary medicine.
Claims33
An adjustable therapeutic gene therapy system for the delivery to, for example, any one of the tissues or cells provided herein, such as to the subcutaneous space (e.g., to one or more cell types in the subcutaneous space), which system comprises:
- a. a genetic construct encoding at least one therapeutic gene, under the regulation of at least one promoter element and, optionally, one or more regulatory elements,
- b. a delivery carrier or vector encapsulating at least part of said genetic construct, optionally, which allows for the re-administration of a subsequent dose of the genetic construct (e.g., at an efficiency of at least 10% as compared to an initial dose of the genetic construct), and
- c. a means of increasing or decreasing the level of therapeutic transgene expression from the genetic construct.
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A method of therapeutic transgene expression comprising, administering a genetic construct encoding at least one therapeutic gene, under the regulation of at least one promoter element and, optionally, one or more regulatory elements, and, optionally, a delivery carrier or vector encapsulating at least part of said genetic construct, to any one of the tissues or cells provided herein, such as to the subcutaneous space (e.g., to one or more cell types in the subcutaneous space).
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Dependencies are inferred from the claim text.