General information

General Topic: Gene Therapy
More specific Topic : Gene Therapy towards Cystic Fibrosis
Research Question: In what way does gene therapy heal such diseases like Cystic Fibrosis?

 a. In brief describe about this application (what is it all about?) 

•  Gene Therapy is a certain method which include genes to be able to prevent and treat various diseases. 
• The therapy itself is defined as a replacement of mutant allele through insertion of genes towards one individual cell. 
• A gene that is responsible for diseases development .
• (rapidly growing field of medicine)
•  Gene therapy is still an experimental discipline and much research remains to be done before this approach to the treatment of condition will realise its full potential
• In gene therapy, only body (somatic) cells and not the egg or sperm cells (germ cells) are targeted for treatment. 

 b. What are some benefits of this application? 

•  Give someone a chance of normal life.
• give chances to someone who was born with a genetic disease by replacing non functional gene with a functional one.
• more efficient
• Very effective when delivered to tissue correctly.
• can avoid drug side effects.
• fixes the problem as its source.
• eliminate and prevent hereditary diseases.

c. What are some future improvements which still need to be done to make this application perfect (limitations)? 

•  Short-lived nature of gene therapy – Sustaining any long-term benefits from DNA integrated into the body is difficult because of the rapidly dividing nature of many cells. Multiple rounds of gene therapy are often needed for any type of cure.
• Immune response – The body often recognizes the vector that is carrying the altered genes as a foreign substance and produces antibodies to attack it
• Problems with viral vectors – There are a variety of potential problems with using inactivated viral vectors: the possibility of immune and inflammatory responses, difficulty in gene control and targeting specific tissues, and the potential of viruses to retain, acquire, or recover pathogenicity (the ability to cause disease).
• Multigene disorders – With so much still unknown about the nature and treatment of multigene disorders, single gene disorders have the best chance of being corrected through gene therapy. However, many common diseases such as heart disease, high blood pressure, arthritis, and diabetes may be caused by multiple gene interactions (polygenic diseases). Unfortunately, until our technology and understanding of the genetic components of these diseases improves, they cannot be treated using gene therapy

d. What issues (social, economic, ethical, political or cultural) are currently related to the use of this application worldwide? 

• Gene Therapy is very expensive
•  Studies for the purposes for which it is used like cosmetic correction.
• use as trait enhancement and neo-eugenics .
• use of genetic science for human good and the avoidance of preventable harms.
• the control and confidentiality of results of genetic testing
• possible discrimination against those found to have genetic diseases 
• Less practical, but possibly more significant, dilemmas involve the use of genetic science in ways that go beyond prevention and cure of disease.
• Justice and resource allocation 
• person’s access to reproductive technologies and privacy rights may conflict with the profit-motive of insurance companies
• people who are concerned by scientists taking the role of God.