Monday 20 October 2014

How effective is Gene Therapy towards Cystic Fibrosis? (Benefits and Limitations)

Original paragraph:   
Gene therapy offers the best hope for a life-saving treatment by tackling the root cause of CF, rather than only treating the symptoms (Cystic Fibrosis Foundation, 1998). The basic concept behind gene therapy is to identify the defective gene and to correct the defect with a normal gene. There are two forms of gene therapy that may be used as a method of treatment. The first is called germ line gene therapy. This form not only helps the individual being treated, but also his or her children as well. Germ line therapy would change the genetic pool and future generations would have to live with that change (Genentech Inc., 1998). The other form of therapy is called somatic gene therapy. Somatic means of, relating to or affecting the body. This therapy involves changing the defective gene in the individual but the change won't be inherited by the next generation. Somatic gene therapy would be the therapy of choice since it doesn't have the ethical considerations that germ line therapy creates.

The obvious con of gene therapy involves the germ line therapy. This therapy not only changes the gene expression for the individual but it also affects following generations. No one knows what this will do to the gene pool that exists afterwards. Another con would be that researchers aren't sure how long the results of gene therapy will work. They also don't know how often the therapy would have to be repeated to get results. As of now, the technology being used isn't adequate enough for gene therapy to be used in people for a range of diseases. All gene therapy at this stage is experimental and cannot be used for any disease. 


A pro of gene therapy is that it doesn't cover up what's wrong but fixes the 'problem'. It has the capability of producing a normal life for the individual that receives the therapy. Another pro is that gene therapy is a fast growing field of its own and more and more is learned about it every day. The technology is there to develop it, so the technology is there to bring it to life. When gene therapy can be actively used, it will be regulated to prevent unnecessary treatment that may be harmful to the patient. Part of the Hippocratic Oath states, "that I will lead my life and practice my art in uprightness and honour; that into whatsoever house I shall enter, it shall be for the good of the sick to the utmost of my power...that I will exercise my art solely for the cure of my patients".


 Key ideas:


  • Gene Therapy offers the best hope for a life-saving treatment by tackling the root cause of Cystic Fibrosis.
  • Methods of treatment: Germ Line Gene Therapy (change of genetic pool) ,Somatic Gene Therapy (change in defective gene but the change won't be inherited by next generation).
  • Basic concept: identify the defective gene and to correct the defect with a normal gene. 


  • Cons (Limitation):
  1. Involves germ line therapy , the therapy changes the gene expression which affects following generations but no one knows what the effect would do to the gene pool afterwards.
  2. Researches aren't sure how long the results of gene therapy will work and how often the therapy would have to be repeated.
  3.       All gene Therapy at this stage is experimental.
  4. Technology being used isn't adequate enough for gene therapy to be used in people for a range of diseases.
  •       Pros (Benefit):

  1.       Fixes the problem.
  2.       It has the capability of producing a normal life.
  3.       Fast growing field.
  4.       Technology is there to bring it to life.
  5.       Prevent unnecessary treatment.


Source in MLA:

"Cystic Fibrosis and Gene Therapy." Cystic Fibrosis and Gene Therapy. N.p., n.d. Web. 19 Oct. 2014. <http://www.ndsu.edu/pubweb/~mcclean/plsc431/students98/senn.htm>.


Paraphrasing:
By taking charge of the source that cause Cystic Fibrosis, Gene therapy provides an aspiration for a life-saving treatment. With a basic concept of identifying a certain defected gene and correcting them with a normal gene , two methods were stated. Germ Line Gene Therapy is one out of the two methods that changes the genetic pool which will then affect upcoming generations while Somatic Gene Therapy exchange the defected gene, yet these changes would not affect generations later. Through the capability of gene therapy in curing Cystic Fibrosis, benefits as well as limitations were considered. Correspondingly , Gene Therapy fixes the disease itself in a way the expression was arranged which then creates an ability to produce a disease-free life. As the therapy develops day by day, new ways referring to the main idea was found to enable improvements for better understanding and an increase in ways to cure. Since gene therapy is one of the treatments considered, patients who received Cystic Fibrosis may prevent harmful unnecessary treatment given due to high technology used. Other than benefits , limitations were figured during the process.A limitation which includes Germ Line Therapy creates a result which was unable to be resolve since the therapy changes the expression and will be affected to following generations giving unknown aftereffect to gene pool afterwards. Researches also weren't positive on the amount of time needed to be able to receive the result and how much repetition is done to be able to succeed.In some occasions, gene therapy is still experimental in a way these technologies is not sufficient. Nevertheless, there are still uncertainties in applying gene therapy into individuals. 




The video uncovers more about the factors preventing gene therapy from being effective.
https://www.youtube.com/watch?v=2G3FS61sbck



No comments:

Post a Comment