HERPES VIRUS-BASED COMPOSITIONS AND METHODS OF USE IN THE PRENATAL AND PERINATAL PERIODS

摘要

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.