C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/86 (2006.01) A61K 48/00 (2006.01) C07K 14/805 (2006.01) C12N 15/85 (2006.01) C12N 15/867 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2164953
A process and means for the design and the optimization of retroviral vectors transducing human .beta.-globin gene and .beta.-Locus Control Region (.beta.-LCR) derivatives, hereafter referred to as C9-globin/LCR] retroviruses, which successfully meet the following criteria required for gene therapy applications: (1) stability of proviral transmission (low frequency of rearrangements similar to retroviral vectors considered stable in the art) upon infection of cell-lines and murine bone marrow cells, (2) improved viral titer, thereby allowing successfful infection of bone marrow cells, and (3) high erythroid expression of the transduced human .beta.-globin gene, are described along with specific constructs.
L'invention concerne un procédé et un moyen de conception et d'optimisation de vecteurs rétroviraux de transduction de dérivés de la région témoin de beta -locus ( beta -LCR) et d'un gène de beta -globine, ci-après dénommés rétrovirus [ beta -globine/LCR], satisfaisant les critères suivants requis par des applications en thérapie génique: (1) stabilité de la transmission provirale (faible fréquence de réagencements similaires aux vecteurs rétroviraux considérés stables dans l'art) lors de l'infection de lignées cellulaires et de cellules de moelle osseuse murine, (2) titre viral amélioré, permettant ainsi une infection réussie de cellules de meolle osseuse, et (3) expression érythroblastique élevée du gène de beta -globine humain transduit. L'invention concerne également des structures spécifiques.
Leboulch Philippe
London Irving M.
Tuan Dorothy
Bereskin & Parr Llp/s.e.n.c.r.l.,s.r.l.
Massachusetts Institute Of Technology
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