FINAL POST

Research Question:
In what way does gene therapy heal such disease like Cystic Fibrosis?

"THE GENE THERAPY ERA" 

In general , Cystic Fibrosis is an inherited disease nor genetic disorder that is caused by a faulty gene that is a mutation on chromosome 7 which disturbs the usual function of epithelial cells.  From findings, around 70,000 worldwide populations are diagnosed with Cystic Fibrosis and they are mainly inherited through genes from neither one nor both of our parents. Epithelial cells are the kind of cells that build up each of its sweat glands in skin and line the passageways in various systems. Normally, the inherited Cystic Fibrosis gives instructions to the body to produce a certain protein called CFTR (cystic fibrosis transmembrane conductance regulator) which creates sweat, saliva , tears , mucus and enzymes. However, if there are complications in the CFTR, these cells may not control the amount of sodium chloride giving troubles to the balance between salt and water reducing its ability to produce thin coating fluid and mucus. Effects may vary in different parts of the organs such as sinuses in nose, sweat glands that creates salty sweat in skin, complication in reproductive organs, blockage in pancreatic or biliary ducts and bacterial infection in airways. Apart from that, with the thick, sticky mucus covering parts such as the lungs, liver, pancreas, and digestive system, it has provided a much more less functional organs and systems.

FIGURE 1
The picture represents the differences of a normal lung airway and a lung airway which is affected by Cystic Fibrosis. For the normal airway, the presence of the CFTR channel is there with precise amount of water,mucus and chloride needed while the lung airway with cystic fibrosis has no presence in CFTR channel that affects the mucus giving a sticky mucus which traps bacteria causing problems in the body.http://www.nchpeg.org/nutrition/index.php?option=com_content&view=article&id=468&tmpl=component


Through a technique called gene therapy, researches are truly convinced that it has the capability to attack diseases that are inherited. For a disease such as Cystic Fibrosis, scientists have discovered a harmless virus known as Adeno-associated virus (AAV) as one of the solution considered since viruses was one of the main method hoping it could replace affected cells of such sickness into healthy ones. Adeno-associated virus is a non-pathogenic virus, which contaminates neither humans nor animals. However, the use of AAV helps transport the genes into cells. As stated, the average life expectancy of a person who received Cystic Fibrosis is 31 where human trials were now done in a procedure, which includes fatty substances as well as liposomes as a path to lead the certain virus and deliver new genes. In result, individuals whom received tests for cystic fibrosis were then free from the disease due to its effectiveness and its ability to be easily detected by our body's natural defence systems. However, we may not know how this treatment may impact various individuals with different body situations.       

FIGURE 2
The pictures shows the gene therapy using an adenovirus vector. The modified gene is inserted between viral genes where during the process, the gene is injected to the certain cell and is then building up proteins with the new gene inserted.
http://www.lysosomalstorageresearch.ca/Fabry_eClinic/images/Content/Management/gene_therapy.jpg

FIGURE 3

The picture describes the process of gene therapy through Adeno-associated virus.The virus itself can't replicate by themselves and therefore require a helper virus such as adenovirus . Also they are non-pathogenic (not known to cause disease).The process itself is also an insertion of genes which will then expresses proteins.

http://cisncancer.org/research/images/gene_therapy_002-w.jpg

By taking charge of the source that causes 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 or to be able to heal. 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 advanced technology used in the techniques.

FIGURE 4
The picture represents chromosome 7 which is a chromosome that is deleted with people who inherited the sickness of Cystic Fibrosis.The mutation itself could effect in many ways such as the loss of amino acids, the lost of protein making ,short in chloride,water as well as mucus and etc.https://www.msu.edu/~luckie/cfsa2.gif

Other than benefits, limitations were figured during the process. A limitation, which includes Germ Line Therapy, creates a result, which was unable to be resolved since the therapy changes the expression and will be affected to following generations. These changes as well are adjustments giving differences in traits of the certain person and with less understanding towards the upcoming product, neither negative nor positive outcome gives 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 as a reason for a few, numerous trails are needed to be completed to succeed since the body might have difficulties in identifying the new gene and rejects after that. Risks in inserting the new gene may also cause harmful DNA mutations having damages in parts of the body and cells inside. 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.

 

FIGURE 5
The picture defines the inheritance of cystic fibrosis in both parents (carriers). Within 4 child in result of a mix of both heterozygous, 25% is affected (homozygous recessive) , 50% carrier (heterozygous)and 25% unaffected (homozygous dominant). Therefore there are 1 in 4 chance the offspring will be affected by cystic fibrosis.
http://upload.wikimedia.org/wikipedia/commons/3/3e/Autorecessive.svg



With Gene Therapy known as a complicated scientific procedure, wide ranges of techniques are expanded to be appropriately used. Considering Gene Therapy as a method that changes its gene structure, many ethical concerns were discussed. It has been told that in some situations, patients who goes through gene therapy still continued their medical drug treatments which give its people second thoughts on how effective the scientific solution is. Sadly, arguments between the solution used for curing diseases nor as an enhance in trait occurred with a reason of fear that gene therapy may be used more as a trait enhancement which by then could badly effect the society and developments in discrimination rises towards people who did inherit unwanted traits. Confusions also enlarge difficulties in comparing traits that are examined as a disease and traits that defines mentally, physiologically and physically. In addition, large amount of ethicists doubt how useful the solution is as well as concerned on how might these genes later be used for future experiments involving genes. As a matter of fact, debates questioning the problems of gene therapy were pursued to permit newer advances that will limit implications regarding assorted factors. 

In conclusion, Gene Therapy did provide beneficial outcomes for Cystic Fibrosis patients and have let new ideas for prevention in such diseases. Even though Gene therapy may own drawbacks, the treatment itself have continuously boost in progress with a point of view that becomes better throughout years of research and experiments. Due to a potentially positive end result, not only it will give possibilities in ending unacceptable inherited sickness, suitable gene therapy treatment of various specific methods will be introduced with a goal to avoid and heal the disease of Cystic Fibrosis.

RESOURCES:

ONLINE:

"Cystic Fibrosis." KidsHealth - the Web's Most Visited Site about Children's Health. Ed. Floyd R. Livingston Jr. The Nemours Foundation, 01 Apr. 2014. Web.  Oct. 2014. <http://kidshealth.org/parent/medical/digestive/cf.html>.

"Cystic Fibrosis Sufferers Offered Hope by Gene Therapy Treatment." The Telegraph. Telegraph Media Group, 28 Sept. 0016. Web. Oct. 2014. <http://www.telegraph.co.uk/health/healthnews/4639764/Cystic-fibrosis-sufferers-offered-hope-by-gene-therapy-treatment.html>.

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

"Ethical and Social Issues in Gene Therapy." Ethical and Social Issues in Gene Therapy. N.p., n.d. Web.  Oct. 2014. <http://www.genetherapynet.com/ethical-and-social-issues-in-gene-therapy.html>.

"FAQs about the Cause, Diagnosis, Treatment of Cystic Fibrosis & More | CF Foundation." FAQs about the Cause, Diagnosis, Treatment of Cystic Fibrosis & More | CF Foundation. N.p., n.d. Web.  Oct. 2014. <http://www.cff.org/AboutCF/Faqs/>.

"Future Therapies and Cystic Fibrosis." Future Therapies and Cystic Fibrosis. N.p., n.d. Web.  Oct. 2014. <http://www.cfmedicine.com/htmldocs/CFText/newtherapy.htm>
"Gene Therapy Adeno-Associated Virus (AAV) Vectors Explained." Gene Therapy Adeno-Associated Virus (AAV) Vectors Explained. N.p., n.d. Web. Oct. 2014. <http://www.genetherapynet.com/viral-vectors/adeno-associated-viruses.html>.

OFFLINE:

THE USBORNE INTRODUCTION TO GENES & DNA :INTERNET LINKED

Author: Anna Claybourne

Editor: Felicity Brooks

Illustrator:Stephen Moncrieff
December 1,2006
DNA AND GENETIC ENGINEERING (CELLS AND LIFE)
Author:Robert Sneeden
August 25,2007

Ethical implications of using Gene Therapy

Original Paragraph:

• The biology of human gene therapy is very complex, and there are many techniques that still need to be developed and diseases that need to be understood more fully before gene therapy can be used appropriately. In addition, because gene therapy involves making changes to the body’s genetic setup, it raises many unique ethical concerns. This clinical was considered successful because it greatly improved the health and well-being of the few individuals who were treated during the trial. However, the success of the therapy was tentative, because along with the gene therapy the patients also continued receiving their traditional drug therapy. This made it difficult to determine the true effectiveness of the gene therapy on its own, as distinct from the effects of the more traditional therapy.
  
  Measuring the success of treatment is just one challenge of gene therapy. Research is fraught with practical and ethical challenges. As with clinical trials for drugs, the purpose of human gene therapy clinical trials is to determine if the therapy is safe, what dose is effective, how the therapy should be administered, and if the therapy works.
  
  Unfortunately, the distinction between gene therapy for disease genes and gene therapy to enhance desired traits, such as height or eye color, is not clear-cut. No one would argue that diseases that cause suffering, disability, and, potentially, death are good candidates for gene therapy. However, there is a fine line between what is considered a "disease" and what is considered a "trait" in an otherwise healthy individual. Even though gene therapy for the correction of potentially socially unacceptable traits, or the enhancement of desirable ones, may improve the quality of life for an individual, some ethicists fear gene therapy for trait enhancement could negatively impact what society considers "normal" and thus promote increased discrimination toward those with the "undesirable" traits. As the function of many genes continue to be discovered, it may become increasingly difficult to define which gene traits are considered to be diseases versus those that should be classified as physical, mental, or psychological traits.
  
  To date, acceptable gene therapy clinical trials involve somatic cell therapies using genes that cause diseases. However, many ethicists worry that, as the feasibility of germ line gene therapy improves and more genes causing different traits are discovered, there could be a "slippery slope" effect in regard to which genes are used in future gene therapy experiments. Specifically, it is feared that the acceptance of germ line gene therapy could lead to the acceptance of gene therapy for genetic enhancement. Public debate about the issues revolving around germ line gene therapy and gene therapy for trait enhancement must continue as science advances to fully appreciate the appropriateness of these newer therapies and to lead to ethical guidelines for advances in gene therapy research.

Key ideas:

• Gene Therapy is very complex, techniques are still developed to be appropriately used.
• Success of Gene Therapy was tentative.
• Traditional drug therapy is still used after gene therapy treatment.
• Ethicists are afraid that gene therapy for trait enhancement could negatively impact what society considers "normal".
• Discrimination towards people who inherited undesirable traits.
• Argues of gene therapy being used to cure diseases or used to enhance desired traits.
• People are worried that genes will be used in experiments.

Source in MLA:

"Ethical and Social Issues in Gene Therapy." Ethical and Social Issues in Gene Therapy. N.p., n.d. Web. 21 Oct. 2014. <http://www.genetherapynet.com/ethical-and-social-issues-in-gene-therapy.html>.

Paraphrasing:

With Gene Therapy known as a complicated scientific procedure, wide ranges of techniques are expanded to be appropriately used. Considering Gene Therapy as a method that changes its gene structure, many ethical concerns were discussed. It has been told that in some situations, patients who goes through gene therapy still continued their medical drug treatments which give its people second thoughts on how effective the scienctific solution is. Sadly, arguments between the solution used for curing diseases nor as an enhance in trait occurred with a reason of fear that gene therapy may be used more as a trait enhancement which by then could badly effect the society and developments in discrimination rises towards people who did inherit unwanted traits. Confusions also enlarge difficulties in comparing traits that are examined as a disease and traits that defines mentally, physiologically and physically. In addition, large amount of ethicists doubt how useful the solution is as well as concerned on how might these genes later be used for future experiments involving genes. As a matter of fact, debates questioning the problems of gene therapy were pursued to permit newer advances that will limit implications regarding assorted factors.

The video explains social as well as ethical consideration regarding to gene therapy in particular.
https://www.youtube.com/watch?v=XowV39JF8eY

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

Gene therapy for Cystic Fibrosis

Original paragraph:   

       Scientists found that a laboratory-created "super" virus could be used to deliver a healthy version of a gene into cells affected by cystic fibrosis, ridding them of the disease.

The discovery is the latest boost for a technique called gene therapy, which scientists hope can attack diseases at their root.

Previous studies have shown that scientists can "turn off" the faulty gene which causes cystic fibrosis by replacing it with a healthy copy.

Tests on human lung cells with cystic fibrosis showed that they were free from the disease after being treated with the virus.

Viruses are one of two key ways that scientists hope they can carry healthy genes into sufferer's cells.

Human trials have already begun into the other method in which liposomes, natural fatty substances which stick to the outside of cells and encourage viruses to enter them, are used to transfer the genes.

The disease, which affects the lungs and causes breathing difficulties, has few known treatments and average life expectancy is just 31.

The scientists modified a harmless virus, known as adeno-associated virus (AAV), which is carried by 90 per cent of the population, into a highly infectious form several hundred times stronger than normal.

The new virus is more effective at entering lung cells than its natural ancestor and better at evading becoming detected by the body's natural defence systems.

The researchers now hope to test the newly created virus in the first ever animal models of the disease, pigs bred last year, and then in humans.

The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene.

Although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient.”

 Key ideas:

•  Scientists found a virus that could be used a new healthy gene towards the cells that are affected by cystic fibrosis
• Tests on human lung cells showed that they were free from the disease after being treated with the virus.
• Viruses are the key scientists hope can carry healthy genes
• average life expectancy is 31
• they have created a modified virus known as adeno-associated virus (AAV)
• The virus is effective at entering lung cells and better at becoming detected by the body's natural defence systems.  
• The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene.

The picture describes its procedure on how gene therapy is used to cure such diseases and how new healthy genes are transmitted into the body . 
http://www.bio.miami.edu/dana/pix/gene_therapy.jpg

Source in MLA:

"Cystic Fibrosis Sufferers Offered Hope by Gene Therapy Treatment." The Telegraph. Telegraph Media Group, 28 Sept. 0016. Web. 14 Oct. 2014. <http://www.telegraph.co.uk/health/healthnews/4639764/Cystic-fibrosis-sufferers-offered-hope-by-gene-therapy-treatment.html>.

Paraphrasing:

Through a technique called gene therapy , researches are truly convinced that it has the capability to attack diseases that are inherited. For a disease such as Cystic Fibrosis, scientists have discovered a harmless virus known as adeno-associated virus (AAV) as one of the solution considered since viruses was one of the main method hoping it could replace affected cells of such sickness into healthy ones. As stated, the average life expectancy of a person who received Cystic Fibrosis is 31 where human trials were now done in a procedure which includes fatty substances as well as liposomes as a path to lead the certain virus and transport new genes. In result, individuals whom received tests for cystic fibrosis were then free from the disease due to its effectiveness and its ability to be easily detected by our body's natural defence systems. However , we may not know how this treatment may impact various individuals with different body situations.

 The interview talks in detail about gene therapy as a possible treatment.

  https://www.youtube.com/watch?v=oC0q7s2udkQ

Introduction of Cystic Fibrosis

Original paragraph:   

Cystic fibrosis (CF) is a genetic disorder that particularly affects the lungs and digestive system.

CF affects more than 30,000 kids and young adults in the United States. It disrupts the normal function of epithelial cells — cells that make up the sweat glands in the skin and that also line passageways inside the lungs, liver, pancreas, and digestive and reproductive systems.

The inherited CF gene directs the body's epithelial cells to produce a defective form of a protein called CFTR (or cystic fibrosis transmembrane conductance regulator) found in cells that line the lungs, digestive tract, sweat glands, and genitourinary system.

When the CFTR protein is defective, epithelial cells can't regulate the way that chloride (part of the salt called sodium chloride) passes across cell membranes. This disrupts the essential balance of salt and water needed to maintain a normal thin coating of fluid and mucus inside the lungs, pancreas, and passageways in other organs. The mucus becomes thick, sticky, and hard to move.

Normally, mucus in the lungs traps germs, which are then cleared out of the lungs. But in CF, the thick, sticky mucus and the germs it has trapped remain in the lungs, which become infected.

 Key ideas:

•  Cystic Fibrosis is a genetic disorder.
• It disrupts the normal function of epithelial cells.
• Inherited CF gene produce a defective form of protein called CFTR (Cystic Fibrosis Conductance Regulator)
• When CFTR is defective,epithelial cells can't regulate sodium chloride. 
• Problems towards the balance of salt and water needed.
• Mucus become thick,sticky and hard to move.
• Mucus that is trapped in certain organ which may become infected.

 The picture shows differences between a normal airway and an airway with cystic fibrosis as well as the affect to various organs.

http://www.nhlbi.nih.gov/health/health-topics/images/cysticfibrosis01.jpg

Source in MLA:

"Cystic Fibrosis." KidsHealth - the Web's Most Visited Site about Children's Health. Ed. Floyd R. Livingston Jr. The Nemours Foundation, 01 Apr. 2014. Web. 06 Oct. 2014. <http://kidshealth.org/parent/medical/digestive/cf.html>.

Paraphrasing:

In general , Cystic Fibrosis is an inherited disease nor genetic disorder that is caused by a faulty gene which disturbs the usual function of epithelial cells. Epithelial cells are cells that builds up the sweat glands in skin and lines passageways in various systems . Normally , the inherited Cystic Fibrosis gives instructions to the body to produce a certain protein called CFTR(cystic fibrosis transmembrane conductance regulator) which creates sweat , saliva , tears , mucus and enzymes. However, if there are complications in the CFTR, these cells may not control the amount of sodium chloride giving troubles to the balance between salt and water reducing its ability to produce thin coating fluid and mucus. Apart from that ,with the thick, sticky mucus covering parts such as the lungs, liver, pancreas, and digestive system , it has provided a much more less functional organs and systems.

More about Cystic Fibrosis:

Videos mainly explains more about cystic fibrosis in detail and how the inherited disease impact individuals.
https://www.youtube.com/watch?v=FMAOEOmLoUE
https://www.youtube.com/watch?v=WuI72eMrIQI&list=PLJBkk17XfXmlRZAGbEqflDw_8tIsuE7RS

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.