Novel internal ribosome entry site and vector containing same

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/86 (2006.01) A61K 31/70 (2006.01) A61K 35/12 (2006.01) A61K 48/00 (2006.01) C12N 15/867 (2006.01)

Patent

CA 2258490

The invention concerns the use of a nucleotide sequence derived from all or part of the genomic RNA 5' end of a type C retrovirus except for Friend murine leukaemia virus (FMLV) and Moloney murine leukaemia virus (MoMLV) as internal ribosome entry site or as element enabling or improving retrovirus vector packaging. The invention also concerns a vector comprising said nucleotide sequence, a viral particle generated from this vector, a cell comprising this vector or infected by the viral particle, their therapeutic use and a pharmaceutical composition containing them. The invention further concerns the use of a vector, a viral particle or a pharmaceutical composition for transfecting or infecting pluripotent stem cells.

La présente invention concerne l'utilisation d'une séquence nucléotidique dérivée de tout ou partie de l'extrémité 5' de l'ARN génomique d'un rétrovirus de type C à l'exception des virus de la leucémie murine de Friend (FMLV) et de Moloney (MoMLV) à titre de site interne d'entrée des ribosomes ou d'élément permettant ou améliorant l'encapsidation de vecteurs rétroviraux. Elle a également pour objet un vecteur comprenant ladite séquence nucléotidique, une particule virale générée à partir de ce vecteur, une cellule comprenant ce vecteur ou infectée par la particule virale, leur utilisation thérapeutique ainsi qu'une composition pharmaceutique les comprenant. Enfin, elle a trait à l'utilisation d'un vecteur, d'une particule virale ou d'une composition pharmaceutique pour la transfection ou l'infection de cellules pluripotentes.

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