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Combined Adeno-Associated Virus And Adenovirus Cocktail Gene Delivery System For High Efficiency Gene Expression Without Eliciting Immune Response In Immuno-Competent Subjects

Summary

The Present Invention Provides An Efficient Gene Delivery System Using Adeno-Associated Viral (Aav) Vector In Gene Therapy. Furthermore, The Invention Provides A Combined Aav And Adenovirus (Adv) Cocktail Gene Delivery System Which Is Even More Efficient In In Vivo Gene Delivery And Expression Without Eliciting Any Significant Immune Responses In An Immunocompetent Subject. In Particular, The Invention Provides A Therapeutic Agent And Methods For Preventing, Treating, Managing, Or Ameliorating Various Diseases And Disorders Including, But Not Limited To, Bone Diseases, By Delivering Bone Morphogenetic Protein 2 (Bmp-2) For New Bone Formation Via Gene Therapy Using Said System. The Invention Provides A Nucleic Acid Molecule Comprising An Avv Vector And A Promoter Operably Linked To A Sequence Encoding Bmp-2; And A Nucleic Acid Molecule Comprising An Adv Vector And A Promoter Operably Linked To A Sequence Encoding Bmp-2, As Well As Vectors And Host Cells Comprising Said Nucleic Acid Molecules, Respectively.

Supplementary Information

Patent Number: US20040223953A1
Application Number: US2004801648A
Inventor: Kung, Hsiang Fu | Chen, Yan | Luk, K.D.K. | Lin, Marie, C.M.
Priority Date: 17 Mar 2003
Priority Number: US20040223953A1
Application Date: 17 Mar 2004
Publication Date: 11 Nov 2004
IPC Current: A61K003818 | A61K004800 | A61P001900 | A61P003500 | C12N001512 | C12N001533 | C12N0015861 | C12N0015864
US Class: 4240932 | 435325 | 435456
Title: Combined adeno-associated virus and adenovirus cocktail gene delivery system for high efficiency gene expression without eliciting immune response in immuno-competent subjects
Usefulness: Combined adeno-associated virus and adenovirus cocktail gene delivery system for high efficiency gene expression without eliciting immune response in immuno-competent subjects
Summary: The nucleic acid molecule is useful for preventing, treating, managing, or ameliorating various diseases and disorders including bone diseases. They are also useful for preventing, treating, managing, or ameliorating various tumors and cancers in a subject. Preferably, the nucleic acid molecule is useful for promoting bone formation, wound healing, tissue repair, nerve cell regeneration, neuronal survival; to stimulate neuronal growth, neural crest cell differentiation, hematopoiesis; to regulate follicle stimulating hormone, lung, and kidney cell function; to decrease proliferation of kidney epithelial cells, proliferation of lung epithelial cells; to inhibit kidney tubule formation; to repair cartilage defects; to prevent stroke; and to suppress the development of gonadal tumors.
Novelty: New nucleic acid molecule comprises an adeno-associated viral vector and a promoter operably linked to a sequence encoding bone morphogenetic protein, useful for preventing, treating, or ameliorating bone diseases or tumors

Industry

Biomedical

Sub Group

DNA/Gene Engineering

Application No.

US2004801648A

Country/Region

Hong Kong

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