Compartmentalized method of nucleic acid delivery and compositions and uses thereof
AssigneeGlobal Bio Therapeutics, Inc.
InventorsJose Gustavo Cabrera Aquino, Blanca Angelica Segura Pacheco
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Abstract
Provided herein is a method of delivering nucleic acid molecules to a compartmentalized tissue or organ of a subject. Also provided herein are uses and processes for delivering a nucleic acid molecule to the parenchyma of a compartmentalized tissue or organ. The methods and uses can be used in the treatment of diseases and conditions and in industrial, agricultural and veterinary applications. Also provided herein are compositions containing an adenovirus or adeno-associated virus or other recombinant virus formulated for administration to the parenchyma of a tissue or organ.
Claims67
A method of delivering a nucleic acid molecule to a subject, comprising:
- a) compartmentalizing a tissue or organ or portion of the tissue or organ from systemic circulation of the host, wherein the portion is sufficient in size to compartmentalize and to effect delivery of the agent;
- and
- b) administering a delivered agent directly to the parenchyma of the tissue or organ or portion of the tissue or organ, wherein the delivered agent comprises the nucleic acid molecule, whereby the delivered agent enters parenchymal cells of the compartmentalized tissue or organ or portion of the tissue or organ.
A container, comprising an adenovirus or adeno-associated virus composition, wherein:
- the composition is formulated for direct parenchymal administration;
- the adenovirus or adeno-associated virus comprises a heterologous nucleic acid molecule in its genome;
- the amount of adenovirus or adeno-associated virus in the composition is an amount for single dosage administration that is 100-fold or less than the amount of the same adenovirus or adeno-associated virus administered intravenously for the same purpose;
- and
- the container is sterile and sealed.
Dependencies are inferred from the claim text.