Gene Therapy Essay

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Recommended: The controversy of gene therapy

Introduction
Gene therapy is a technique which has the prospect of providing an advantageous method in curing genetic diseases. Although still in development, successful trials have occurred, and conceptually, the idea being tested is ideal in the fight against lethal diseases such as haemophilia, cystic fibrosis and cancer. However, much controversy surrounds the idea, due to its undeveloped nature, potential biological risks and hefty failures in the past. The use and development of gene therapy in the future is a deliberated topic. This investigation aimed to consider potential risks and benefits, giving rise to the question: gene therapy; friend or foe?
Biological Background
Gene therapy is a process used to cure diseases and disorders caused by mutated or defective genes in patients, which alters or inhibits the production of essential proteins used in normal functionality (Hunt, 2008). Fundamentally, the treatment involves the delivery of functioning DNA into cells, which incorporates into the genome and replaces the defective gene, or repairs, alters or deactivates genes causing the disease (Genetic Home Reference, 2014; Hunt, 2008).
Gene therapy employs vectors to deliver functioning genes into cells, a ‘vector’ being a vehicle in which DNA can be transferred. They can be viral or non-viral, and treatment can occur in vitro and ex vivo (inside and outside the body) (ScienceDaily, 2014; University of Utah, 2014). The use of viral vectors mimics the survival techniques of viruses. A virus is a miniscule particle containing genetic material; lacking material to reproduce on its own, it infects a host, injecting its DNA or RNA, and utilises the host’s cellular machinery to reproduce (Freudenrich, 2014). In gene therapy,...

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...lestaple (2011), BBC Horizon Trial and Error, the Rise and Fall of Gene Therapy, accessed 18th May 2014,
 TrinsicAnimation (2011), Gene Therapy Medical Animation (CERE-120), accessed 8TH June 2014,
 University of Utah (2014), Approaches to Gene Therapy, accessed 25th May 2014
 University of Utah (2014), Gene Delivery: Tools of the Trade, accessed 25th May 2014
 University of Utah (2014), Gene Therapy Successes, accessed 25th May 2014,
 Viral and Non-viral Vectors in Gene Therapy:
 Wikipedia (2014), Gene Therapy, accessed 25th May 2014,

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