公开(公告)号：EP1036177A1

公开(公告)日：2000-09-20

申请号：EP98960546.4

申请日：1998-11-25

申请人： THE IMMUNE RESPONSE CORPORATION

发明人： ILL, Charles, R. ,  GONZALES, Jose, E., N. ,  YANG, Claire, Q. ,  BIDLINGMAIER, Scott

IPC分类号： C12N15/12 ,  C12N15/67 ,  C12N15/85 ,  C07K14/755 ,  A61K48/00

CPC分类号： C07K14/755 ,  A61K48/00 ,  C12N15/67 ,  C12N15/85 ,  C12N2830/008 ,  C12N2830/30 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2830/85 ,  C12N2840/44 ,  C12N2840/445

摘要： Novel genes and vectors exhibiting increased expression and novel splicing patterns are disclosed. The gene can comprise one or more consensus or near consensus splice sites which have been corrected. The gene can alternatively or additionally comprise one or more introns within coding or noncoding sequences. The gene can still further comprise modified 5' and/or 3' untranslated regions optimized to provide high levels and duration of tissue-specific expression. In one embodiment, the gene comprises the coding region of a full-length Factor VIII gene modified by adding an intron within the portion of the gene encoding the β-domain, so that the gene is expressed as a β-domain deleted Factor VIII protein. The novel Factor VIII gene can also be modified to correct one or more consensus or near consensus splice sites within or outside of the coding region.

公开(公告)号：EP0888451A1

公开(公告)日：1999-01-07

申请号：EP97908930.0

申请日：1997-03-10

申请人： THE IMMUNE RESPONSE CORPORATION

发明人： ILL, Charles, R. ,  BIDLINGMAIER, Scott

IPC分类号： C12N15 ,  C07K14 ,  C12N9

CPC分类号： C12N9/644 ,  C07K14/755 ,  C12N15/85 ,  C12N2800/108 ,  C12N2830/42 ,  C12N2830/48 ,  C12N2840/44 ,  C12Y304/21022

摘要： Expression vectors are disclosed comprising intronless genes containing one or more near consensus splice sequences and one or more copies of a viral cis-acting post-transcriptional regulatory element which is transcribed along with the gene and causes export of the gene transcript from the nucleus into the cytoplasm of the cell. In a preferred embodiment, the vectors are targeted for delivery to specific cells in the form of a molecular complex made up of the plasmid releasably linked to a nucleic acid binding agent and a ligand which binds to a component on the surface of a cell. Use of viral cis-acting post-transcriptional regulatory elements as disclosed can increase expression of intronless genes with near-consensus splice sites.