EP 1833975 A2 20070919 - REGULATED EXPRESSION OF TRANSGENES IN THE CENTRAL NERVOUS SYSTEM OF MAMMALS
Title (en)
REGULATED EXPRESSION OF TRANSGENES IN THE CENTRAL NERVOUS SYSTEM OF MAMMALS
Title (de)
REGULIERTE EXPRESSION VON TRANSGENEN IM ZENTRALNERVENSYSTEM VON SÄUGERN
Title (fr)
EXPRESSION REGULEE DE TRANSGENES DANS LE SYSTEME NERVEUX CENTRAL DE MAMMIFERES
Publication
Application
Priority
- US 2005044654 W 20051209
- US 63535604 P 20041209
Abstract (en)
[origin: WO2006063247A2] Recombinant adeno-associated virus (rAAV) vectors are provided for delivery of regulatable transgenes to the central nervous system (CNS) of a mammal. Also provided are methods of treatment of subjects with neurodegenerative disorders using the vectors, and kits for constructing or using the vectors or performing the methods of the invention. Transgene sequences are expressed from a promoter/enhancer region comprising one or more binding sites for a transcription factor that is responsive to a small molecule inducer. Both the transgene construct and a construct comprising the transcription factor are delivered to the target cells. The regulatable transgene may be delivered on the same rAAV vector as the transcription factor, or on a separate vector. The transcription factor may comprise two polypeptide chains, e.g. a DNA binding domain and a transcription activation domain, that form an active dimer in the presence of a dimerizer such as rapamycin or a non-immunogenic analog thereof. Vectors, methods and kits of the invention may be used to deliver genes such as AADC or GDNF to the brain of a subject with a neurodegenerative disorder, such as Parkinson's disease, where the expression of AADC or GDNF in the brain can subsequently be regulated by treatment of the subject with rapamycin or a rapamycin analog.
IPC 8 full level
C12N 15/864 (2006.01); A61K 35/76 (2015.01); A61K 48/00 (2006.01); A61P 25/16 (2006.01)
CPC (source: EP)
A61P 25/00 (2018.01); A61P 25/16 (2018.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/00 (2013.01); C12N 2830/42 (2013.01); C12N 2830/85 (2013.01); C12N 2840/203 (2013.01)
Designated contracting state (EPC)
AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LI LT LU LV MC NL PL PT RO SE SI SK TR
DOCDB simple family (publication)
WO 2006063247 A2 20060615; WO 2006063247 A3 20060803; CN 101213305 A 20080702; EP 1833975 A2 20070919; IL 183580 A0 20070920; JP 2008523093 A 20080703; MX 2007006807 A 20071115
DOCDB simple family (application)
US 2005044654 W 20051209; CN 200580042455 A 20051209; EP 05849656 A 20051209; IL 18358007 A 20070531; JP 2007545670 A 20051209; MX 2007006807 A 20051209