ATE524554T1 - Nicht integrativer und nicht replikativer lentivirus, herstellung und verwendungen - Google Patents

Nicht integrativer und nicht replikativer lentivirus, herstellung und verwendungen

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Publication number
ATE524554T1
ATE524554T1 AT05779704T AT05779704T ATE524554T1 AT E524554 T1 ATE524554 T1 AT E524554T1 AT 05779704 T AT05779704 T AT 05779704T AT 05779704 T AT05779704 T AT 05779704T AT E524554 T1 ATE524554 T1 AT E524554T1
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Austria
Prior art keywords
integrative
production
vivo
nervous system
central nervous
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AT05779704T
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English (en)
Inventor
Jacques Mallet
Che Serguera
Stephanie Philippe
Chamsy Sarkis
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Centre Nat Rech Scient
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AT05779704T 2004-06-25 2005-06-24 Nicht integrativer und nicht replikativer lentivirus, herstellung und verwendungen ATE524554T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR0407017A FR2872170B1 (fr) 2004-06-25 2004-06-25 Lentivirus non interactif et non replicatif, preparation et utilisations
PCT/FR2005/001604 WO2006010834A1 (fr) 2004-06-25 2005-06-24 Lentivirus non integratif et non replicatif, preparation et utilisations

Publications (1)

Publication Number Publication Date
ATE524554T1 true ATE524554T1 (de) 2011-09-15

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AT05779704T ATE524554T1 (de) 2004-06-25 2005-06-24 Nicht integrativer und nicht replikativer lentivirus, herstellung und verwendungen

Country Status (10)

Country Link
US (1) US8119119B2 (de)
EP (1) EP1761635B1 (de)
JP (1) JP4861314B2 (de)
CN (1) CN101023177A (de)
AT (1) ATE524554T1 (de)
AU (1) AU2005266221B2 (de)
CA (1) CA2579753C (de)
FR (1) FR2872170B1 (de)
IL (1) IL179740A0 (de)
WO (1) WO2006010834A1 (de)

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EP1761635B1 (de) 2011-09-14
CA2579753C (fr) 2015-04-14
IL179740A0 (en) 2007-05-15
FR2872170A1 (fr) 2005-12-30
CN101023177A (zh) 2007-08-22
WO2006010834A1 (fr) 2006-02-02
AU2005266221B2 (en) 2010-06-10
US8119119B2 (en) 2012-02-21
CA2579753A1 (fr) 2006-02-02
FR2872170B1 (fr) 2006-11-10
JP2008503230A (ja) 2008-02-07
US20080089863A1 (en) 2008-04-17
AU2005266221A1 (en) 2006-02-02
JP4861314B2 (ja) 2012-01-25

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