Adenoviral vectors for gene delivery in skeletal muscle...

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/86 (2006.01) A61K 38/00 (2006.01) A61K 48/00 (2006.01) C07K 14/075 (2006.01) C12N 15/00 (2006.01) C12N 15/62 (2006.01) C12N 15/861 (2006.01)

Patent

CA 2423045

The invention provides means and methods for transduction of a skeletal muscle cell and/or a muscle cell specific precursor thereof. Provided is the use of a gene delivery vehicle derived from an adenovirus, having a tropism for said cells, for the preparation of a medicament. In a preferred aspect of the invention, said gene delivery vehicle comprises at least a tropism determining part of an adenoviral fiber protein of subgroup B and/or F. More preferably, said gene delivery vehicle comprises at least part of a fiber protein of an adenovirus of stereotype (11, 16, 35, 40 and/or 51) or a functional part, derivative and/or analogue thereof.

L'invention concerne des moyens et procédés de transduction d'une cellule de muscle squelettique et/ou d'un précurseur spécifique de cette cellule de muscle. L'invention concerne également l'utilisation d'un véhicule d'apport de gène, dérivé d'un adénovirus et possédant un tropisme à l'égard de ces cellules, dans la préparation d'un médicament. Dans un aspect préféré de l'invention, ce véhicule d'apport de gène comprend au moins une partie déterminante du tropisme d'une protéine fibreuse adénovirale du sous-groupe B et/ou F, et dans un aspect encore plus préféré, ce véhicule comprend au moins une partie d'une protéine fibreuse d'un adénovirus de sérotype 11, 16, 35, 40 et/ou 51, ou une partie, un dérivé et/ou un analogue, fonctionnels, de celui-ci.

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