In utero somatic gene therapy in the later stages of pregmaternal liver. Expression of the -galactosidase transnancy may allow targeting of organ systems which are difgene was detected in many fetal tissues by RT-PCR. High ficult to reach later in life and to prevent the development -galactosidase production was shown by immuno-histoof tissue damage otherwise caused by the early onset of chemistry predominantly in the liver, where about 30% of inherited diseases. We report here on the percutaneous the hepatocytes stained positive, and in the adrenal cortex. delivery of two adenoviral vectors, containing the -Production of factor IX was determined by ELISA in the galactosidase reporter gene and the human Factor IX gene plasma of treated fetuses and newborn lambs and reached respectively, to the fetal liver and circulation by ultrasoundat birth up to 80% of the normal human plasma concenguided umbilical vein puncture similar to procedures used tration. This demonstrates a very hopeful proof of principle in human pregnancy. Vector spread, as detected by PCR for the development of prenatal treatment of many genetic analysis for the -galactosidase encoding vector, was diseases but also requires more detailed investigations found in almost all fetal and neonatal organs and in the with respect to the observed systemic spread of the vector.
As ultrasound-guided amniocentesis in humans is a well-established diagnostic procedure, delivery of the factor IX gene into the amniotic cavity appears to be a safe route for prenatal treatment of haemophilia B and may prevent haemorrhagic complications such as intracranial bleeding during delivery. Our study allowed for the first time a quantification of the expression of a potentially therapeutic transgene in rodents after prenatal gene delivery. It thus provides a model for the prenatal treatment of haemophilia B, but may also serve as a pathfinder to gene therapy of inheritable skin disorders such as epidermolysis bullosa.
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