Gene Delievery System

If a child or an embryo is diagnosed to carry a defective gene leading to disability, one may like to correct this defect by any of the following three methods.  (i) by replacement of defective gene with a normal gene (ii) by correcting the defective gene through gene targeting (iii) by gene augmentation either through increasing the no. of copies of the gene or through a higher level of expression of the introduced gene .  Such a correction of a genetic defect is described as gene therapy.  The system would be useful to some of the potential diseases like Haemophilia, HBV, Diabetes, Cancer for which therapeutics is being developed. Maximum progress in the area of gene therapy has been achieved through the gene augmentation method involving vector mediated DNA delivery system. US Patent has been granted on the process.