Herpes virus-based compositions and methods of use in the...

A - Human Necessities – 61 – K

Patent

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A61K 48/00 (2006.01) A61K 35/76 (2006.01)

Patent

CA 2615690

Disclosed are compositions and methods for reducing the severity of a birth defect in a mammal by exposing the mammal (e.g., in utero) to a herpes virus amplicon particle comprising a cis element-flanked transgene and a sequence encoding a transposase. Upon expression, the transposase inserts the transgene into the genome of a cell (e.g., a neuron) within the mammal and the transgene expresses a polypeptide or RNA that compensates for a protein or gene defect that is causally associated with the birth defect.

L'invention concerne des compositions et des procédés de réduction de la gravité d'une anomalie à la naissance chez un mammifère consistant à exposer le mammifère (par exemple in utéro) à une particule d'amplicon de virus herpétique contenant un transgène flanqué d'un élément cis et une séquence codant une transposase. Lors de l'expression, la transposase insère le transgène dans le génome d'une cellule (par exemple un neurone) à l'intérieur du mammifère et le transgène exprime un polypeptide ou un ARN qui compense une anomalie protéique ou génique associée par effet de causalité à l'anomalie à la naissance.

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