Gene therapy at Royal Free Hospital London
Prof Ted Tuddenham & Prof. Amit Nathwani are leading the research in both haemophilia A & B In the UK. Prof Tahira Zafar , Director HTC Rawalpindi met them recently to discuss Gene Therapy for haemophilia patients
Genes carry Instructions to make proteins which perform most life functions. Altered Genes produce abnormal proteins which are unable to carry normal functions resulting in Genetic disorders. Gene therapy is insertion of normal genes to replace an abnormal gene Into an individual’s cells and tissues to treat a disease. Gene therapy is being studied in clinical trials (research studies with people) It is not currently available outside clinical trials.
Haemophilia is especially well suited for gene therapy because the clinical manifestations are attributable to the lack of a single protein that circulates in minute amounts in the plasma
Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in hemophilia A and B. Patient data supports sustained endogenous production of clotting factor as a result of gene therapy . This eliminates the need for infusion of coagulation factors
Although gene therapy is a promising treatment option for a number of diseases the technique is being intensively worked at to make sure that it will be safe and effective.