Hung Long Do

US Patent:

6517830, Feb 11, 2003

Filed:

Aug 4, 2000

Appl. No.:

09/633020

Inventors:

John S. Lollar - Decatur GA
Hung V. Do - Atlanta GA
John F. Healey - Snellville GA
Edmund K. Waller - Atlanta GA

Assignee:

Emory University - Atlanta GA

International Classification:

A01N 6300

US Classification:

424 9321, 514 44, 4353201

Abstract:

Compositions and methods are provided for the in vivo gene delivery of nucleic acid sequences encoding the factor VIII protein to the liver endothelial sinusoidal cells (LSECs). Compositions and methods are also provided for the ex vivo gene transfer of nucleic acid sequences encoding the factor VIII protein to cultured LSECs and the implantation of the transformed LSECs in vivo. These methods and compositions increase the level of factor VIII in the blood stream and find use in the gene therapy treatment of hemophilia A.

US Patent:

20030129174, Jul 10, 2003

Filed:

Dec 3, 2002

Appl. No.:

10/308637

Inventors:

John Lollar - Decatur GA, US
Hung Do - Atlanta GA, US
John Healey - Snellville GA, US
Edmund Waller - Atlanta GA, US

Assignee:

Emory University

International Classification:

A61K048/00
C12N005/08
C12N015/867
C12N015/861

US Classification:

424/093210, 435/456000, 435/366000

Abstract:

Compositions and methods are provided for the in vivo gene delivery of nucleic acid sequences encoding the factor VIII protein to the liver endothelial sinusoidal cells (LSECs). Compositions and methods are also provided for the ex vivo gene transfer of nucleic acid sequences encoding the factor VIII protein to cultured LSECs and the implantation of the transformed LSECs in vivo. These methods and compositions increase the level of factor VIII in the blood stream and find use in the gene therapy treatment of hemophilia A.