Gene therapy is used for

Gene transfer is a novel area of therapeutics in which the active agent is a nucleic acid sequence rather than a protein or small molecule. Most gene transfers are carried out using a vector or gene delivery vehicle because delivery of naked DNA or RNA to a cell is an inefficient process. More clear-cut success has been achieved in a gene therapy trial for another form of SCID, adenosine deaminase (ADA) deficiency. Other diseases likely to be amenable to transduction of hemaopietic stem cells (HSCs) include • Wiskott-Aldrich syndrome • Chronic granulomatous disease • Sickle cell disease • Thalassemia. Clinical trials using recombinant adeno-associated vectors are now ongoing for muscular dystrophies, alpha-1 antitrypsin deficiency, lipoprotein lipase deficiency, hemophilia B, and a form of congenital blindness called Leber’s congenital amaurosis.