Gene Therapy Vs Germline Therapy

Gene therapy has a long rugged road ahead before it can be widely accepted in society. Gene therapy is an experimental technique in which genes are manipulated to prevent disease. Doctors hope to use this technique in the future to prevent future genetic disorders by injecting a gene in the patient's cells instead of having to turn to surgery or drugs. Currently this process is only used if all other therapies are rendered ineffective. The two most frequently used methods are somatic therapy and germline therapy. In germline therapy genes are modified in the sperm or ova and when they fuse into a zygote, the zygote then can, during division, pass the modified gene to all the other cells during the development of the offspring. As a result,
this change is also passed to future generations.On the other hand, somatic therapy only affects the individual being treated. In this process DNA is inserted into body cells; usually a virus is used to deliver fixed or missing genes. These two therapies show great promise in treating diseases such as parkinson's disease, alzheimer's, and hemophilia, however there are still many studies required to make sure these therapies are safe and effective.

Parkinson's disease is a disorder in the central nervous system, which affects movement, including tremors.
According to professor Nicholas Mazarakis,the Lucas Lee Chair of Molecular Biomedicine at Imperial College London, the treatment is called Prosavin and like a virus this treatment delivers three genes into the stratum that increase levels of dopamine which is not produced in patients with parkinson's disease.This is only logical since the striatum is the area of the brain that controls movement. After doing human trials on 15 people in advanced stages of the disease Mazarakis noticed that patients movement tests had increased up to 30 percent and that patients reported having a better quality of life. However, the study had no placebo so the results are interpreted with caution. Even though this gene therapy experiment does not offer concrete results it is certainly ready for the next
phase.
The use of gene therapy has also been used to slow down the Alzheimer's disease. The trial was lead by Mark Tuszynski, MD, PhD, from the University of California in san diego. in this trial five woman and three men in the early stages of alzheimer's were followed for about 2 years. At the start of the trial the scientist took scans and tests o f the brain and also at regular intervals so they could see the gradual process of change. Tuszynski approach consisted of taking skin cells called fibroblasts from each of the patients and genetically modifying the cells to secrete nerve growth factor, which, is a protein that prevents nerve cell death and stimulates the brain. According to Tuszynski, that decline of mental activity was about half as slow after the procedure. Even though this gene therapy was proved to slow alzheimer's and not cure it, the finding has the potential to be great.

Gene therapy has also shown great potential in people with the disease hemophilia.
Hemophilia is a medical condition in which the blood’s ability to clot is high reduced, causing an individual to bleed abundantly even with the slightest cut. The original replacement therapy was to repetitive and carried to much risks so the St. jude's children's research hospital and the university college of london lead a team to find a new approach for this disease. The scientists used the factor IX and modified into a virus that targets livers cells since the liver is the only place where the factor IX can be produced for the clotting process. According to the new england journal of medicine men before the study the 6 men were producing less than 1 percent of the factor IX, but after ranged from 2 to 11 percent and after 6 to 16 months 4 of the 6 men no longer need factor IX infusions and others needed it less often. Indeed this therapy shows promising
results.

conclusion
Whether gene therapy is something good or bad it will bring many about many social, economic and scientific implications. If gene therapy is done successfully without damaging an individual or terribly affecting the generations to come, people will question the idea that gene therapy can only be used as a last resort. The health industry will find a way to make big profit from gene therapy. Moreover, this knowledge will allow scientists to uncover to new ways of treating other diseases with gene therapy.