Synthetic mecp2 sequence for protein substitution therapy

Details Synthetic mecp2 sequence for protein substitution therapy Synthetic mecp2 sequence for protein substitution therapy

C - Chemistry, Metallurgy

07

K

C07K 14/46 (2006.01)

Patent

CA 2647125

The invention relates to the MeCP2 protein and its use in protein substitution therapy. More specifically, the invention relates to codon-optimized nucleic acid sequences for the expression of MeCP2 proteins, methods for creating such a nucleic acid sequence and expressing such a protein, fusions of a protein of the invention to a transduction domain, and vectors and host cells comprising a protein of the invention. Further, the invention relates to uses of nucleic acids or proteins of the invention in medicine, pharmaceutical compositions comprising nucleic acid sequences and proteins of the invention, as well as methods for the treatment, prevention, and/or therapy of neurodegenerative or neurodevelopmental diseases including Rett syndrome.

L'invention concerne la protéine MeCP2 et son utilisation dans une thérapie de substitution protéique. Plus spécifiquement, l'invention concerne des séquences d'acides nucléiques à codon optimisé permettant l'expression des protéines MeCP2, des procédés de création d'une telle séquence d'acides nucléiques et d'expression de ladite protéine, des fusions d'une protéine de l'invention avec un domaine de transduction, ainsi que des vecteurs et des cellules hôtes contenant une protéine de l'invention. L'invention concerne en outre les utilisations des acides nucléiques ou des protéines de l'invention dans des médicaments, des compositions pharmaceutiques contenant les séquences d'acides nucléiques et les protéines de l'invention, de même que des procédés de traitement, de prévention et/ou de thérapie de maladies neurodégénératives ou neurodéveloppementales, y compris le syndrome de Rett.

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