Method of treating type I diabetes using an AAV vector encoding uracortin 2

Patent metadata

Field	Value
Publication number	US-10918738-B2
Application number	US-201816233970-A
Country	US
Kind code	B2
Filing date	Dec 27, 2018
Priority date	Feb 14, 2012
Publication date	Feb 16, 2021
Grant date	Feb 16, 2021

Abstract

In alternative embodiments, the invention provides methods for treating, ameliorating or protecting (preventing) an individual or a patient against a disease, an infection or a condition responsive to an increased paracrine polypeptide level in vivo comprising: providing a paracrine polypeptide-encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence; or an expression vehicle, a vector, a recombinant virus, or equivalent, having contained therein a paracrine-encoding nucleic acid or gene, and the expression vehicle, vector, recombinant virus, or equivalent can express the paracrine-encoding nucleic acid or gene in a cell or in vivo; and administering or delivering the paracrine polypeptide-encoding nucleic acid or gene operatively linked to a transcriptional regulatory sequence, or the expression vehicle, vector, recombinant virus, or equivalent, to an individual or a patient in need thereof, thereby treating, ameliorating or protecting (preventing) the individual or patient against the disease, infection or condition responsive to an increased paracrine polypeptide level.

First claim

What is claimed is:

1. A method for treating type 1 diabetes in a mammal comprising: injecting an adeno-associated viral vector (AAV) comprising a nucleic acid sequence encoding Uracortin 2 (UCN2) operably linked to a promoter to a mammal with type 1 diabetes intravenously (IV) such that increased insulin release and decreased hyperglycemia in the mammal occurs, thereby treating the type 1 diabetes in the mammal.

2. The method of claim 1 , wherein the vector is selected from AAV8 or AAV9.

3. The method of claim 1 , wherein the promoter is selected from a chicken beta-actin (CBA), thyroid hormone-binding globulin (TBG), or Rous Sarcoma Virus (RSV).

4. The method of claim 1 , wherein the mammal is a human.

5. The method of claim 1 , wherein the mammal is a canine, non-human primate or feline subject.

6. The method of claim 1 , wherein the method comprises between about 1 to 5 intravenous (IV) injections.

7. The method of claim 1 , wherein the vector is AAV-DJ or AAVrh10.

8. The method of claim 1 , wherein the adeno-associated viral vector (AAV) is formulated as a buffered aqueous solution.

9. The method of claim 1 , wherein the adeno-associated viral vector (AAV) is formulated in a liposome.

10. The method of claim 9 , wherein the liposome is a multilayered liposome.

11. The method of claim 1 , wherein the adeno-associated viral vector (AAV) is formulated in a nanoparticle or nanolipoparticle.

Assignees

• Univ California

Inventors

• Hammond H Kirk
• Gao Mei Hua

Classifications

• A61K38/2228 Primary

  Corticotropin releasing factor [CRF] (Urotensin) · CPC title

• A61K47/6901

  Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors · CPC title

• A61K2121/00

  Preparations for use in therapy · CPC title

• A61P21/00

  Drugs for disorders of the muscular or neuromuscular system · CPC title

• A61K48/005

  characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title