Target cell-specific vectors for inserting genes into cells,...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/85 (2006.01) A61K 31/70 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01) C12N 15/87 (2006.01) C12N 15/88 (2006.01)

Patent

CA 2197265

Target cell-specific vectors for inserting genes into cells, pharmaceuticals comprising such vectors and their use Target cell-specific vectors for inserting at least one gene into cells of an organism, which vectors comprise the following components: a) a non-viral carrier for the gene to be inserted, b) a ligand which can bind specifically to the desired target cell, c) a fusion protein for the penetration of the vector into the cytoplasm of the target cell, and d) the gene to be introduced are disclosed. Vectors of this nature are used, in particular, in gene therapy.

Vecteurs spécifiques d'une cellule cible pour l'insertion de gènes dans les cellules, produits pharmaceutiques comprenant ces vecteurs, et leur utilisation. Sont décrits dans la présente des vecteurs spécifiques d'une cellule cible qui permettent d'insérer au moins un gène dans les cellules d'un organisme, vecteurs composés des éléments suivants : a) un support non-viral pour le gène à insérer, b) un ligand capable de se lier spécifiquement à la cellule cible désirée, c) une protéine de fusion pour la pénétration du vecteur dans le cytoplasme de la cellule cible, et d) le gène à insérer. Les vecteurs de cette nature servent notamment en thérapie génique.

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