EP 2173765 A2 20100414 - ADENO-ASSOCIATED VIRAL VECTORS FOR THE EXPRESSION OF DYSFERLIN
Title (en)
ADENO-ASSOCIATED VIRAL VECTORS FOR THE EXPRESSION OF DYSFERLIN
Title (de)
ADENO-ASSOZIIERTE VIRUSVEKTOREN ZUR EXPRESSION VON DYSFERLIN
Title (fr)
VECTEURS VIRAUX ADÉNO-ASSOCIÉS POUR L'EXPRESSION DE LA DYSFERLINE
Publication
Application
Priority
- FR 2008051414 W 20080725
- FR 0705479 A 20070726
Abstract (en)
[origin: WO2009016326A2] The present invention relates to a composition comprising: a first adeno-associated viral (AAV) vector comprising: i) a 5'ITR (Inverted Terminal Repeat) sequence of AAV; ii) a portion of gene placed under the control of a promoter; iii) a sequence comprising a splice donor site; iv) a 3'ITR sequence of AAV; and/or a second adeno-associated viral (AAV) vector comprising: v) a 5'ITR (Inverted Terminal Repeat) sequence of AAV; vi) a sequence comprising a splice acceptor site; vii) a portion of gene; viii) a 3'ITR sequence of AAV. The combination of the portions of gene carried by the first and second AAV vectors comprises an open reading frame which encodes a functional dysferlin. In addition, the combination of the sequence comprising the splice donor site and the sequence comprising the splice acceptor site contains all the elements necessary for the splicing, advantageously derived from a natural intron of the dysferlin gene.
IPC 8 full level
C07K 14/47 (2006.01)
CPC (source: EP US)
A61P 19/00 (2017.12 - EP); A61P 21/00 (2017.12 - EP); C07K 14/4707 (2013.01 - EP US); C12N 15/86 (2013.01 - EP US); A61K 48/00 (2013.01 - EP US); C12N 2750/14143 (2013.01 - EP US)
Citation (search report)
See references of WO 2009016326A2
Designated contracting state (EPC)
AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MT NL NO PL PT RO SE SI SK TR
Designated extension state (EPC)
AL BA MK RS
DOCDB simple family (publication)
FR 2919305 A1 20090130; FR 2919305 B1 20090918; CA 2697127 A1 20090205; EP 2173765 A2 20100414; US 2010266551 A1 20101021; WO 2009016326 A2 20090205; WO 2009016326 A3 20090430
DOCDB simple family (application)
FR 0705479 A 20070726; CA 2697127 A 20080725; EP 08826741 A 20080725; FR 2008051414 W 20080725; US 67045108 A 20080725