C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/85 (2006.01) A61K 48/00 (2006.01) A61P 3/00 (2006.01) C12N 15/00 (2006.01) C12N 15/62 (2006.01) C12N 15/63 (2006.01) C12N 9/12 (2006.01) C12N 15/54 (2006.01)
Patent
CA 2719711
A composition comprising: a stably integrating delivery vector; an modified mammalian thymidylate kinase (tmpk) activator polynucleotide wherein the modified mammalian tmpk polynucleotide encodinges a modified mammalian tmpk polypeptide that increases phosphorylation of converts a prodrug relative to phosophorylation of the prodrug by wild-type mammalian tmpk polypeptideto a drug; and/or a targeting polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent. The disclosure also relates to use of these compositions in methods of treatment of diseases such as Fabry disease.
L'invention concerne une composition comprenant : un vecteur d'administration intégrant de façon stable; un polynucléotide activateur de la thymidylate kinase (tmpk) de mammifère modifié, le polynucléotide de tmpk de mammifère modifié codant pour un polypeptide tmpk de mammifère modifié qui augmente la phosphorylation ou convertit en un médicament un promédicament relativement à la phosphorylation du promédicament par un polypeptide tmpk de mammifère de type sauvage; et/ou un polynucléotide ciblant codant pour un polypeptide de surface cellulaire qui se lie sélectivement à un agent de liaison toxique. L'invention porte également sur l'utilisation de ces compositions dans des procédés de traitement de maladies, telles que la maladie de Fabry.
Bereskin & Parr Llp/s.e.n.c.r.l.,s.r.l.
University Health Network
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