Transgenic animals produced using oral administration of a genetic agent coupled to a transporting agent

• US 20060030540A1
• Filed: 10/06/2005
• Published: 02/09/2006
• Est. Priority Date: 07/18/2002
• Status: Abandoned Application

• Current Assignee: NeoX, Inc.
• Original Assignee: NeoX, Inc.
• Inventors: Hortelano, Gonzalo, Vargas, Andrew Gomez
• Application Number:
  US11/244,782
• Publication Number:
  US 20060030540A1
• Time in Patent Office: Days
• US Class Current: 514/44.R
• CPC Class Codes: A01K 2217/05 Animals comprising random inserted nucleic acids (transgenic)
• CPC Class Codes: A01K 67/0271 Chimeric vertebrates, e.g. comprising exogenous cells
• CPC Class Codes: A01K 67/0275 Genetically modified vertebrates, e.g. transgenic
• CPC Class Codes: A61K 47/61 the organic macromolecular compound being a polysaccharide or a derivative thereof
• CPC Class Codes: A61K 47/645 Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
• CPC Class Codes: A61K 47/6927 the form being a solid microparticle having no hollow or gas-filled cores
• CPC Class Codes: A61K 47/6929 the form being a nanoparticle, e.g. an immuno-nanoparticle
• CPC Class Codes: A61K 48/0025 wherein the non-active part clearly interacts with the delivered nucleic acid
• CPC Class Codes: A61K 48/0041 the non-active part being polymeric
• CPC Class Codes: A61K 48/0075 characterised by an aspect of the delivery route, e.g. oral, subcutaneous
• CPC Class Codes: A61P 21/04 for myasthenia gravis
• CPC Class Codes: A61P 3/10 for hyperglycaemia, e.g. antidiabetics
• CPC Class Codes: A61P 31/00 Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
• CPC Class Codes: A61P 31/18 for HIV
• CPC Class Codes: A61P 35/00 Antineoplastic agents
• CPC Class Codes: A61P 43/00 Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
• CPC Class Codes: A61P 7/04 Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
• CPC Class Codes: B82Y 5/00 Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
• CPC Class Codes: C12N 9/644 Coagulation factor IXa (3.4.21.22)
• CPC Class Codes: C12Y 304/21022 Coagulation factor IXa (3.4.21.22)

Abstract

The present invention is directed toward a method of producing transgenic animals by administration of a composition to an animal via a natural gastrointestinal pathway. The composition achieves widespread distribution, systemic expression and sustained delivery in the animal. More particularly, the invention discloses a method for producing a transgenic animal using oral gene therapy. The present invention also provides transgenic animals produced by this method.

Claims

1. An in vivo process for producing a transgenic animal comprising:

providing at least one transporting agent effective for enabling widespread distribution, systemic expression and sustained delivery of at least one genetic material via a natural gastrointestinal pathway, said genetic material designed to express a biological agent;

forming a distributable moiety by coupling said transporting agent and said genetic material;

administering said distributable moiety to an animal;

transporting said distributable moiety in vivo via said natural gastrointestinal pathway whereby said genetic material is included within essentially all cells of said animal; and

genetically altering said animal via intracellular expression of said biological agent;

wherein a transgenic animal is produced.

2. The process in accordance with claim 1 wherein said animal is a wild-type or a transgenic animal.

3. The process in accordance with claim 1 wherein said step of administering is accomplished rectally.

4. The process in accordance with claim 1 wherein said step of administering is accomplished orally and said process further includes, subsequent to forming said distributable moiety, a step of combining said distributable moiety with a protective agent in a manner effective to protect said distributable moiety during traversal of said natural gastrointestinal pathway.

5. The process in accordance with claim 1 wherein said step of expressing is ubiquitous.

7. The process in accordance with claim 1 wherein said genetic material comprises at least one complete transcriptional unit.

8. The process in accordance with claim 1 wherein said transporting agent is a polypeptide.

9. The process in accordance with claim 1 wherein said transporting agent is a compound containing an amine group and constructed and arranged to couple with said genetic material to enable widespread distribution, systemic expression and sustained delivery of said genetic material.

10. The process in accordance with claim 1 wherein said coupling is via electrostatic binding.

11. A transgenic animal produced by the process in accordance with claim 1 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

12. A transgenic animal produced by the process in accordance with claim 2 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

13. A transgenic animal produced by the process in accordance with claim 3 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

14. A transgenic animal produced by the process in accordance with claim 4 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

15. A transgenic animal produced by the process in accordance with claim 5 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

17. A transgenic animal produced by the process in accordance with claim 7 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

18. A transgenic animal produced by the process in accordance with claim 8 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

19. A transgenic animal produced by the process in accordance with. Claim 9 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

20. A transgenic animal produced by the process in accordance with claim 10 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.

6. The process in accordance with clam 1 wherein said step of expressing is tissue specific.

16. A transgenic animal produced by the process in accordance with claim 6 wherein said animal is characterized by a lack of germ cell alteration thereby precluding progenic transmission.