The University of Sheffield is considered one of the most forefront institutions researching cell and gene therapy. Announced earlier this year, The Gene Therapy Innovation and Manufacturing Centre (GTIMC) led by the University’s own Professor Mimoun Azzouz, is one of the three pioneering hubs launching in the UK dedicated to research into cell and gene therapy. The GTIMC is part of a new £18million network funded by LifeArc and the Medical Research Council (MRC); with additional funds coming from the Biological Sciences Research Council.
Despite the UK being a world-class genetics research base, it has long been cited that academics and researchers have experienced logistical problems in their research. By concentrating this research into these institutions, the aim is to eliminate these problems and therefore streamline the research process. The intended downstream response of this is to ‘smooth’ the transition between small-scale supply for early clinical trials through the large-scale development and hopeful manufacture of novel therapies.
What is gene therapy? To begin with, a gene is a segment of a person’s DNA that often codes for a protein. Proteins are incredibly important to the human body, and when mutations occur in the DNA, they can sometimes affect the proteins that segment – that gene – they code for. Sometimes this change is minuscule, sometimes in the case of, for example, Cystic Fibrosis -one of the most infamous genetic disorders), there is no protein produced which has disastrous effects on bodily health. Gene therapy is a way of modifying these defective genes to cure or minimise the symptoms of disease.