GENE THERAPY

Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene to replace a mutated gene. Gene therapy was first conceptualised in 1972, with the authors urging caution before commencing human gene therapy studies. The first attempt, an unsuccessful one, at modifying human DNA was performed in 1980 by Martin Cline. The first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2011, Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia. In 2012, Glybera, a treatment for a rare inherited disorder, lipoprotein lipase deficiency, was approved by the European Commission. Although the technology is still in its infancy, it has been used with some success. It is a potential therapy for a number of diseases (such as cystic fibrosis, sickle cell anaemia, adrenoleukodystrophy and haemophilia) as well as several inherited retinal diseases. Current gene therapy has primarily focused on treating individuals by targeting the therapy to somatic (body) cells, such as bone marrow…