Tabriz University of Medical Sciences Advanced Pharmaceutical Bulletin 2228-5881 3 2 2013 12 30 Gene Therapy, Early Promises, Subsequent Problems, and Recent Breakthroughs 249 255 10.5681/apb.2013.041 EN Saeideh Razi Soofiyani Behzad Baradaran Farzaneh Lotfipour Tohid Kazemi Leila Mohammadnejad Journal Article 10.5681/apb.2013.041 2013 02 13 Gene therapy is one of the most attractive fields in medicine. The concept of gene delivery to tissues for clinical applications has been discussed around half a century, but scientist’s ability to manipulate genetic material via recombinant DNA technology made this purpose to reality. Various approaches, such as viral and non-viral vectors and physical methods, have been developed to make gene delivery safer and more efficient. While gene therapy initially conceived as a way to treat life-threatening disorders (inborn errors, cancers) refractory to conventional treatment, to date gene therapy is considered for many non–life-threatening conditions including those adversely influence on a patient’s quality of life. Gene therapy has made significant progress, including tangible success, although much slower than was initially predicted. Although, gene therapies still at a fairly primitive stage, it is firmly science based. There is justifiable hope that with enhanced pathobiological understanding and biotechnological improvements, gene therapy will be a standard part of clinical practice within 20 years.