Gene therapy – Principles and applications

Abstract

The concept of gene therapy dates back to the 1960s and 1970s and it is yet to come of age, as there is a paucity of reliable, long-term data on the safety and efficacy of this novel therapy. It is fast emerging as a promising approach to treat, cure or prevent genetic based diseases by correction of defective genes that are responsible for disease development. Gene therapy is a key treatment strategy for disorders caused by a missing or faulty gene. It involves introduction of functional genes into appropriate cells so that they can produce sufficient amounts of proteins, encoded by the transferred gene or turn off genes that are causing problems and eventually resulting in permanent correction of the disorder. While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance. Gene therapy treatments are of broadly two types, Germline gene therapy, & Somatic Germline gene therapy. There are two methods for administering gene therapy -Ex vivo & in-vivo. There are two main parts to gene replacement: Genes and Vectors. Gene therapy is useful for hematologic conditions as Hemoglobinopathies, Hemophilia and Inherited bone marrow failure. Future Prospective of Gene Therapy in Transfusion Medicine ,work is being done to explore ex vivo generation of RBCs utilizing various cell fractions, including hematopeitic progenitor cells from cord blood and adult sources. Cost of therapy and ethical issues are the limiting factors for the use of Gene therapy.