Gene therapy with chimeric oligonucleotides delivered by a...

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/64 (2006.01) A01K 67/027 (2006.01) A61K 31/712 (2006.01) A61K 48/00 (2006.01) C12N 15/11 (2006.01) G01N 33/50 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2443923

The present invention provides a method for enhancing the in vivo delivery of chimeric oligonucleotides, containing for example DNA/2'OMeRNA, into cells of a plant, an animal or an human, comprising a step of applying topically to or injecting into a tissue, or tissue adjacent to a tissue, containing said cells, a composition comprising said chimeric oligonucleotide, followed, preceded by, or simultaneous to a step of transferring said chimeric oligonucleotide into said cells by iontophoresis, and relates to a gene therapy method comprising the iontophorically transfer of a chimeric oligonucleotide DNA/2'OMeRNA. The present invention is also directed to particular chimeric oligonucleotides DNA/'OMeRNA capable of inducing or inhibiting the expression of a specific gene involved in eye function by inducing or reverting a mutation in that specific gene, and their use as therapeutic composition for preventing or treating ocular diseases.

L'invention concerne un procédé permettant de renforcer l'administration in vivo d'oligonucléotides chimériques contenant par exemple de l'ADN/2'OMeANR, dans les cellules d'une plante, d'un animal ou d'un humain, qui comprend une étape d'application topique sur un tissu ou d'injection dans ledit tissu ou bien sur ou dans un tissu adjacent, contenant lesdites cellules, une composition comprenant lesdits oligonucléotides chimériques, ladite étape étant suivie ou précédée d'une étape de transfert desdits oligonucléotides chimériques dans lesdites cellules par iontophorèse, ou bien les deux actions étant menées simultanément. L'invention concerne un procédé de thérapie génique comprenant le transfert iontophorique d'un oligonucléotide chimérique ADN/2'MOeARN. Ladite invention concerne en outre des oligonucléotides chimériques particuliers aptes à induire ou à inhiber l'expression d'un gène spécifique impliqué dans la fonction oculaire par induction ou inversion d'une mutation dans ce gène spécifique, ainsi que leur utilisation comme composition pharmaceutique pour prévenir ou traiter des affections oculaires.

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