Kyson X Chou

US Patent:

20100303776, Dec 2, 2010

Filed:

Apr 16, 2010

Appl. No.:

12/799095

Inventors:

Richard Jude Samulski - Chapel Hill NC, US
Kyson Xiaohuai Chou - Irvine CA, US

International Classification:

A61K 35/12
C12N 5/10
C07H 21/02
C12N 15/63
A61K 31/7105
C12P 19/34
A61P 43/00

US Classification:

424 9321, 435325, 536 231, 4353201, 514 44 R, 435 913

Abstract:

The present invention provides a cell comprising a first heterologous nucleic acid construct and a second heterologous nucleic acid construct, wherein each of said first and second heterologous nucleic acid constructs comprises: A. a first nucleotide sequence encoding a nucleotide sequence of interest (NOI); and B. a second intronic nucleotide sequence comprising: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule encoded by said first nucleotide sequence in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining a second intron different from said first intron, wherein said second intron is removed by splicing to produce no RNA molecule and/or to produce a second RNA molecule that is not encoded by said first nucleotide sequence, wherein each said first nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another and wherein each said second intronic nucleotide sequence of each of said first and second heterologous nucleic acid constructs is different from one another.

US Patent:

20100196335, Aug 5, 2010

Filed:

Apr 28, 2006

Appl. No.:

11/919267

Inventors:

Richard J. Samulski - Chapel Hill NC, US
Kyson Xiaohuai Chou - Irvine CA, US

International Classification:

A61K 35/12
C07H 21/02
C12N 15/74
C12N 5/10
A61K 31/7088
C12P 21/00
C12P 19/34
A61K 31/7105
C12Q 1/68
A61P 43/00

US Classification:

424 9321, 536 231, 4353201, 435325, 514 44 R, 435 681, 435 915, 435366, 514 44 A, 435 6

Abstract:

The present invention provides an isolated nucleic acid comprising: a) at least one first nucleotide sequence encoding a heterologous nucleotide sequence of interest; and b) at least two second heterologous nucleotide sequences, wherein each second heterologous nucleotide sequences comprises: i) a first set of splice elements defining a first intron that is removed by splicing to produce a first RNA molecule that imparts a biological function in the absence of activity at a second set of splice elements; and ii) the second set of splice elements defining one or more introns different from said first intron, wherein said one or more introns different from said first intron are removed by splicing to produce no RNA molecule and/or a second RNA molecule that docs not impart a biological function, when said second set of splice elements is active. Further provided are methods of using the nucleic acid of this invention to regulate transgene expression.