Gene Therapy for Rare Diseases

Gene therapy is a logical way to treat rare inheritable conditions; cure a single gene disfigurement by introducing a' correct' gene. The first gene- remedy trials were conducted using cases with rare monogenetic diseases, but these are now outnumbered by the clinical testing of gene therapeutics for more common conditions, such as cancer, AIDS and cardiovascular disease. due to a failure to achieve long- term gene expression with early vector systems, a critical demand for correcting numerous inborn inheritable blights. Now, with the arrival of adeno- associated viral( AAV) and lentiviral vectors, which demonstrate persistent gene expression in animal studies, this technological hedge may have been overcome.

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