Gene Therapy: Gene Delivery Systems And Treatment Of Rare Diseases

A gene therapy medical product is defined by the European Medicines Agency (EMA) as having two characteristics: (a) it comprises an active substance that contains or consists of a recombinant nucleic acid utilized in or provided to human beings with a view to regulating, correcting, replacing, or adding or deleting a genetic sequence; and (b) its therapeutic, prophylactic, or diagnostic impact relates directly to the recombinant nucleic acid. Vaccines against infectious illnesses are not permitted in gene therapy ,however clinical successes in 2009-2011 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber congenital amaurosis X¿linked SCID, ADA¿SCID, adrenoleukodystrophy, and Parkinson¿s disease,