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Gene Therapy

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Megan Harmon

on 30 March 2015

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Transcript of Gene Therapy

The transplantation of normal genes into cells in places of missing or defective ones in order to correct genetic disorders. It has many promises for the future because it has the potential to correct diseased tissues and help organs work properly.
What is Gene Therapy?
Gene Therapy
A vector (viral or non-viral) is genetically engineered to deliver a gene. It is modified by changing the proteins on the surface of the vector. The vector is then injected into the patient.
How it Works
Gene Therapy is used to treat and potentially cure genetic disorders such as cancer, hemophilia and Parkinson's disease.
How it's Used
Scientist inject a vector into the body to potentially modify and hopefully cure the genetic disorder possessed by the patient. The vector's DNA is transcribed and translated in order to create its certain proteins that will attack/fix the diseased tissues.
The Procedure
Vectors are DNA delivery "vehicles". They are used to move certain modified DNA to a patient's cell.
Two Vectors of Gene Therapy
- made with plasmids, circular pieces of double-stranded DNA
-non-viral vector
- made with lipid-based packets (similar to a cell's membrane
- transfer their DNA by fusing to the cell membrane
- very low effectiveness at integrating
- some can be toxic and they typically don't trigger an immune response from the body.
Liposomes
- viral vector
- carries genetic material in the form of RNA
- targets cells that are still dividing
- can be engineered to target certain cell types by changing proteins on the viral surface
- enters the cell and the nucleus, where its RNA can be converted into DNA at random spots in the host
- injection causes immune response

Retroviruses
Because this field has so much potential to treat the disorders we used to think were untreatable, there are many new emerging therapies that can successfully cure or help these disorders.
The future for gene therapy research is relatively bright.
Current Status of Gene Therapy Research
- Once the DNA is placed into the human genome, the cells divide quickly, restricting gene therapy from providing any long term benefits
-They will have to undergo multiple treatments of therapy to continue the benefits.
-Another reason that gene therapy hasn't been successful is because of the response of the patients immune system. When something unfamiliar enters the body, the system's job is to attack.
-In the end, the therapy wouldn't have worked.
- There are also troubles with viral rectors, meaning that once inside the body, the gene therapy may end up causing diseases for the patient.
-Also, because single gene disorders are best for gene therapy,many of the multi gene disorders would not be able to be treated.
-These diseases include Alzheimer's, arthritis, and diabetes.

Factors that Limit Gene Therapy's Effective Treatment
Recently, gene therapies have begun to find success in non-cancerous diseases.
Also, gene targeting has become more efficient and accurate.
The future for a cure for cancer is looking bright. A modified version of the herpes simplex 1 virus(called T-VEC) is being developed to kill only cancer cells.
Recent Developments in Gene Therapy Research
- right now, it has the capability to cure serious genetic disorders such as hemophilia
- further development may help to find a cure for cancer
- gene therapy allows those with genetic disorders to pass the corrected genes to offspring by inserting them into the reproductive cells
Benefits of Using Gene Therapy
Sites
Ethical Considerations
Unknown risks involved
Vectors can be dangerous in many ways
Retroviruses could disrupt surrounding genes and ultimately cause tumors
Body will possibly
not
trigger immune response and result in death
Body will possibly
over
trigger immune response and result in death
Unseen risk that may result in detrimental effects
Problems cannot be estimated until there is harm caused
http://guardianlv.com/2014/07/gene-therapy-benefits-and-potential-risks/

http://ghr.nlm.nih.gov/handbook/therapy/genetherapy

http://www.iptv.org/EXPLOREMORE/ge/uses/use2_gene.cfm

http://learn.genetics.utah.edu/content/genetherapy

http://www.mdanderson.org/patient-and-cancer-information/cancer-information/cancer-topics/cancer-treatment/chemotherapy/gene-therapy/index.html

www.musc.edu/humanvalues/pdf/gene-therapy.pdf
Gene therapy is a fairly costly procedure. According to Santa Clara University, the estimated total cost per person once gene therapy treatments begin may be as high as 100,000. Because of this, it is mainly only accessible to the rich.
Costs and Accessibility
- how can we customize our babies?
- Can we make cows with more than 1 utter?
- CAN WE CURE CANCER?
Additional Questions
Ethical Considerations
Unknown risks involved
Vectors can be dangerous in many ways
Retroviruses could disrupt surrounding genes and ultimately cause tumors
Body will possibly
not
trigger immune response and result in death
Body will possibly
over
trigger immune response and result in death
Unseen risk that may result in detrimental effects
Problems cannot be estimated until there is harm caused
Megan, Audrey, Julian, Olivia, Griffin
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