Juan Li, 38Pittsburgh, PA

2002007, Jun 20, 2002

Apr 20, 2001

09/838786

Liangwu Sun - Pittsburgh PA, US
Juan Li - Pittsburgh PA, US
Xiao Xiao - Wexfoxd PA, US

C12P021/06

435/069100

A method for overcoming the packaging limitations of recombinant adeno-associated virus (AAV) particles through AAV heterodimer formation is provided. In the method, an expressed nucleic acid, typically a portion of a gene encoding a full-length therapeutic protein, or a functional derivative thereof, is split into two or more fragments by the insertion of one or more introns. Each intron is then split and each of the gene portions are inserted between AAV ITRs for packaging into recombinant adeno-associated virus particles. The recombinant viral particles are then co-infected into a target cell. Once inside the cell, the viral vectors form head-to-tail heterodimers through sequence homology of the inverted terminal repeats, thereby re-forming the intron. During mRNA maturation, the intron is spliced from the continuous DNA molecule, removing the intron and, thus, the intervening ITR sequences, thereby restoring the precise coding sequence of the expressed nucleic acid.

2002019, Dec 19, 2002

Aug 14, 2002

10/217568

Xiao Xiao - Wexford PA, US
Richard Samulski - Chapel Hill NC, US
Juan Li - Wexford PA, US

A61K048/00
C12N015/861

424/093200, 435/456000

A method of expressing a gene product in the muscle tissue of an animal, which comprises administering a recombinant AAV vector to the muscle tissue of the animal, wherein the vector comprises a non-AAV gene of interest ligated into an AAV vector genome.

2009029, Dec 3, 2009

Aug 7, 2009

12/537806

Xiao Xiao - Wexford PA, US
Richard J. Samulski - Chapel Hill NC, US
Juan Li - Wexford PA, US

A61K 48/00

514 44 R

A method of expressing a gene product in the muscle tissue of an animal, which comprises administering a recombinant AAV vector to the muscle tissue of the animal, wherein the vector comprises a non-AAV gene of interest ligated into an AAV vector genome.