3. Modificazioni Transgeniche (insertion di nouvi geni)

• Gene therapy can be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene therapy the recipient's genome is changed, but the change is not passed along to the next generation. In germline gene therapy, the parents egg and sperm cells are changed with the goal of passing on the changes to their offspring. 

• Gene Knockout models: Gene knockout to inactivate the Lhx5 gene in the mouse, embryos lacking Lhx5 function developed a malformed hippocampus. For this brain structure to develop normally, cells must first migrate to the correct position in the developing brain and then form the precisely ordered tissue layers of the hippocampus. In the Lhx5 knockout embryos, cells failed to migrate normally, and this resulted in a grossly distorted structure in which regular cell layers could no longer be recognized 

• Transgenic animal models to study disease:  A transgenic rat model for Alzheimer's Disease - use a DNA construct harbouring the Swedish APP 670/671 mutation. The ideal result would be a rat, where expression of human APP with the codon 670/671 mutation gives rise to AD pathology whereas a rat with the normal human APP gene gives no AD pathology. A transgenic rat expressing AD pathology will be of great importance in analysing the pathogenesis and developing treatments for the disease.

• Gene expression systems: Cells in vitro - high yielding cell lines early in cell construction programs can produce vaccines and other proteins e.g. 00-800 mg/liter. Cloning of the cell line results in an improvement of yields. Maximum expression levels attainable depend on the product but cell lines have been created that are capable of producing over 1g of recombinant antibody per litre with specific production rates of typically 20 to 50pg/cell/day. The high cost of manufacture of protein products, particularly of therapeutic proteins, means that maximising yield per cell is essential if economic processes are to be established.