Using Gene Therapy as a treatment for Cystic Fibrosis
Introduction:
Gene therapy is a treatment which deals with a problem at its source, unlike other forms of treatment which merely eliminate the symptoms. Cystic Fibrosis (being a single gene disorder) should, theoretically, be one of the simpler diseases to cure using this method. In this paper Gene Therapy, in relation to the treatment of Cystic Fibrosis, will be discussed, along with its effectiveness and limitations. The process of Gene Therapy using an Adenovirus vector will also be addressed as this is the most common method in the treatment of this disease.
Cystic Fibrosis is a life threatening disorder which is passed on genetically. It is an infection of the secretory glands caused …show more content…
Firstly, Gene Therapy is seen as a short lived solution to a lifetime problem. It is incredibly difficult to sustain long term benefits from integrating DNA into the body because of the rapid pace at which cells divide which leads to multiple round of gene therapy before the disease can be cured. Secondly, the vector carrying the altered genes is often recognized by the body as a foreign substance, and anti-bodies are then produced to attack it. There are also issues which can arrive as a result of using inactivated viral vectors. Some of these issues are as follows: The difficulty in targeting specific genes and the controlling of the genes, the risk of inflammatory and immune responses, and the possibility of the viruses acquiring, retaining, or recovering pathogenicity (the capability of causing disease). Gene Therapy has the best chance of working in the situation of single gene disorders (such as in Cystic Fibrosis), however our limited understanding of the genetic components of multigene disorders (for example arthritis and diabetes) prevent Gene Therapy from being a treatment option for such diseases. Lastly, viruses are capable of infecting more than one type of cell, resulting in the infection of additional cells and not just the targeted cells which contain the mutated genes. On this occasion, healthy cells could be damaged causing other diseases and illnesses, …show more content…
Although it is theoretically simple to access the lungs in an invasive way, the gene transfer into the airways of humans has shown to be practically inefficient, and there is no proof that the gene transfer efficiency is enough to rid the body of the disease all together. It has proved to be a difficult disease to develop an effective gene therapy for, and I personally believe that more testing should be done to ensure the safety of the outcome of the transfer. If science has not proven this method 100% safe, as well as effective, it should be avoided as there could be unknown and harmful symptoms which follow the
Introduction:
Gene therapy is a treatment which deals with a problem at its source, unlike other forms of treatment which merely eliminate the symptoms. Cystic Fibrosis (being a single gene disorder) should, theoretically, be one of the simpler diseases to cure using this method. In this paper Gene Therapy, in relation to the treatment of Cystic Fibrosis, will be discussed, along with its effectiveness and limitations. The process of Gene Therapy using an Adenovirus vector will also be addressed as this is the most common method in the treatment of this disease.
Cystic Fibrosis is a life threatening disorder which is passed on genetically. It is an infection of the secretory glands caused …show more content…
Firstly, Gene Therapy is seen as a short lived solution to a lifetime problem. It is incredibly difficult to sustain long term benefits from integrating DNA into the body because of the rapid pace at which cells divide which leads to multiple round of gene therapy before the disease can be cured. Secondly, the vector carrying the altered genes is often recognized by the body as a foreign substance, and anti-bodies are then produced to attack it. There are also issues which can arrive as a result of using inactivated viral vectors. Some of these issues are as follows: The difficulty in targeting specific genes and the controlling of the genes, the risk of inflammatory and immune responses, and the possibility of the viruses acquiring, retaining, or recovering pathogenicity (the capability of causing disease). Gene Therapy has the best chance of working in the situation of single gene disorders (such as in Cystic Fibrosis), however our limited understanding of the genetic components of multigene disorders (for example arthritis and diabetes) prevent Gene Therapy from being a treatment option for such diseases. Lastly, viruses are capable of infecting more than one type of cell, resulting in the infection of additional cells and not just the targeted cells which contain the mutated genes. On this occasion, healthy cells could be damaged causing other diseases and illnesses, …show more content…
Although it is theoretically simple to access the lungs in an invasive way, the gene transfer into the airways of humans has shown to be practically inefficient, and there is no proof that the gene transfer efficiency is enough to rid the body of the disease all together. It has proved to be a difficult disease to develop an effective gene therapy for, and I personally believe that more testing should be done to ensure the safety of the outcome of the transfer. If science has not proven this method 100% safe, as well as effective, it should be avoided as there could be unknown and harmful symptoms which follow the