The genetics of diseases other than cancer

Gene therapy is a not a new field and has been evolving for decades. Even with the best efforts of researchers around the globe, there has only been limited success in gene therapy mainly with the common diseases like cancer. Will gene therapy overcome the diseases that have no other cures?
Gene therapy is an experimental technique that treats or prevents diseases through the use of genes. Scientists have focused on diseases that are caused by single-gene defects such as sickle cell anaemia, muscular dystrophy, haemophilia and cystic fibrosis. A gene can't easily be directly inserted into your cells, therefore it's delivered using a carrier called a vector. There are unsolved problems with the technology including its short lived nature, human immune response, problems with viral vectors, multigene disorders, inducing tumors, the cost. Although there are risks involved, gene therapy is showing much promise in overcoming the rarer diseases as there have been clinical successes since 2006, these include Leber's congenital amaurosis, adrenoleukodystrophy. haemophilia and Parkinson's disease.
Cystic Fibrosis (CF) is a fatal genetic disease that leads to many health problems (fig.1), also the most common lethal autosomal recessive disease in the worlds Caucasian population. It is caused by a point mutation in the gene cystic fibrosis transmembrane conductance regulator(CFTR). Most people have two working copies (alleles) of the CFTR gene where only one is needed to prevent cystic fibrosis. With modern medicine the life expectancy of people with CF has increased, there is no definite cure for the disease (fig.2). Gene therapy for CF has been tested in humans using liposome's and viruses. At present the effect seen only lasts for a few ...

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...minated at a substantially lower rate than unmodified T-cells, in a larger group of patients as well as plans to increase the persistence of more cells in the body to achieve a therapeutic effect.

Gene therapy has seen many failures and successes, but our knowledge on gene therapy and research into the field has seen leaps in advancements of treatments and therapies. Gene therapy still has a very long way to go due to the dangers of the field, but it definitely has the potential to cure the incurable diseases, due to their having been breakthroughs in early stages of research. Gene therapy has already surpassed many other treatments with effectiveness. When researchers have delved even deeper into this field that is full of potential there is a high probability that incurable diseases will see cures, but it will still be many years of research and understanding.