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US 2020/0345867 A1Published application
Organ directed gene delivery
AssigneeThe Johns Hopkins University
InventorsFlorin M. Selaru, Vivek Kumbhari, Ling Li
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Abstract
Intrabiliary hydrodynamic injection of nucleic acid for in vivo gene therapy for treatment of liver or pancreas disease and other disorders.
Claims25
A method of transfecting cells of a subject in vivo, comprising:
- administering at elevated pressure an effective amount of a nucleic acid expression cassette to the subject's biliary tree, liver, kidney or pancreas.
A method of transfecting cells of a subject in vivo, comprising:
- administering at elevated pressure an effective amount of a nucleic acid expression cassette to the subject,
- wherein the elevated pressure is measured in real-time during and/or subsequent to administering of the nucleic acid expression cassette.
A method of treating or preventing liver or pancreas disease by genetic therapy comprising the steps of:
- delivering a vector comprising a nucleic acid sequence that ameliorates a liver or pancreas disease to a subject having or prone of getting a liver or pancreas disease,
- creating a substantially closed space;
- injecting the vector under required pressure and quantity into the closed space so that the vector is transferred to the cytoplasm of cells of the liver or pancreas;
- expressing the nucleic acid sequence and treating or preventing a liver or pancreas disease in the subject.
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Dependencies are inferred from the claim text.