Gene therapy in current medical arena

Gene therapy is an experimental technique that uses genes encoding the expression of proteins that are either endogenous or biological to treat or prevent a disease by inserting it into patient’s cells. Previously the challenges faced were the gene titers which then changed to their delivery and now expression of the genes. Initially the trial of gene therapy for adenosine deaminase deficiency had proven to be successful but till date only alipogenetiparvovec has been approved in Europe for lipoprotein lipase deficiency. Recent techniques with adenovirus, r-adeno associated virus (r-aav), retrovirus,herpes simplex virus vectors and non-viral lipoplexes (for transfer of large biological products) have completely revolutionized the future of gene therapy. With the upcoming advances in the field of cancer therapy such as herpes simplex thymidine kinase (hs-tk) and its role in suicidal gene therapy in the treatment of cancers has stretched new horizons in the field of gene therapy. Recently the newer treatment approaches with RNA interference and anti-sense oligonucleotides in muscular dystrophies were certainly encouraging for the researchers to further work on the newer prospects of gene therapy. Gene therapy is currently investigated for cardiac failure and for prevention of brain ageing in Alzheimer’s disease.