PRENATAL THERAPY

摘要

Compositions and methods are provided to treat or prevent a genetic disease or disorder, e.g., Hemophilia A, in a subject by administering to the subject an effective amount of a polynucleotide encoding a nucleic acid encoding a functional Factor VIII polypeptide in a pre-term placenta-derived stem cell. The subject to be treated can be a fetus and the cell is a pre-term chorionic villus tissue derived stem cell (CSC) that has been isolated from the placenta before the baby is born.