Herpes Virus-based Compositions and Methods of Use in the Prenatal
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چکیده
Assignee: UNIVERSITY OF ROCHESTER, (57) ABSTRACT Rochester, NY (US) Disclosed are compositions and methods for reducing the Appl. No.: 11A916,434 severity of a birth defect in a mammal by exposing the mam y x9 mal (e.g., in utero) to a herpes virus amplicon particle com PCT Fled: Jun. 5, 2006 prising a cis element-flanked transgene and a sequence encoding a transposase. Upon expression, the transposase PCT NO.: PCT/USO6/21806 inserts the transgene into the genome of a cell (e.g., a neuron) within the mammal and the transgene expresses a polypeptide S371 (c)(1), or RNA that compensates for a protein or gene defect that is (2), (4) Date: Mar. 28, 2008 causally associated with the birth defect.
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