| 摘要 |
Disclosed is the use of a composition for manufacture of a medicament for treating a disease or condition treatable by a therapeutic nucleic acid, wherein: - the composition comprises a delivery agent comprising the therapeutic nucleic acid molecule; - the composition is formulated as suitable for injection of the delivery agent comprising the nucleic acid molecule into the parenchyma of a compartmentalised tissue, organ, or portion of a tissue or organ sufficient in size to compartmentalise and to effect administration of the composition; - the delivery agent is a virus or vector that comprises the therapeutic nucleic acid; - the composition contains 1 x 10^3 to 1 x 10^12 viral particles or vectors; - the composition contains a single dose of the virus or vector; - the parenchyma of a compartmentalised tissue, organ or portion thereof does not include connective tissue, blood vessels, nerves or ducts; - the disease or condition treatable by a therapeutic nucleic acid is selected from among a genetic deficiency, a disease or condition ameliorated by an exogenous gene product, and a disease or condition involving aberrant or altered expression of a gene product in its etiology; and - the compartmentalised tissue or organ or portion of a tissue or organ is isolated from the systemic circulation, wherein the organ is clamped whereby the arteries, veins, ducts and/or vessels servicing or traversing the tissue or organ or portion of the tissue or organ are blocked so that blood supply and flow to the tissue or organ or portion thereof is reduced or eliminated. |
