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Gene Therapy

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by

J Alonzo

on 12 December 2013

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Transcript of Gene Therapy

What is gene therapy?
- is a technique that uses genes to prevent or treat certain types of diseases and disorders

- a process used to correct the effects of defective genes by inserting a normal gene into the body

- may allow doctors to treat diseases without the use of surgery or drugs

- removes a mutated gene that is functioning improperly

- it introduces a new gene into the body to help fight certain diseases
How gene therapy works?
Scientists use a carrier called a vector that is genetically engineered to deliver the gene into the cell. Viruses are usually used as the vector because they are able to deliver the new healthy gene into the cell by infecting it. The viruses are modified so they will not infect the person by a disease during the process.


- the vector is inserted into the body or given through IV
-once introduced into the vector, it infects targeted cells
-the vector unloads its genetic material containing the healthy human gene into the target cell
Importance
-corrects defective genes responsible for disease development

-could possibly be the cure for certain diseases that cannot be cured by drugs or surgery

-can be used to cured cancer, brain diseases (Alzheimer's) and Cystic Fibrosis by replacing faulty genes in the body in hope that this will repair the genes
by: Julia and Jobelle
Types of Gene Therapy
Somatic cell therapy: is a non-reproductive process that affects only the targeted cells in the patient, and is not passed on to future generations. The effects of somatic cell therapy are often short-lived because the cells of most tissues eventually die and are replaced by new cells. Therefore repeated treatments over the course of the patient's life are needed to maintain the therapeutic effect.
Germ line therapy: is a reproductive process that results in permanent changes that are passed down to future generations. If done early during the embryo development, the genetic transfer could also occur in all cells of the developing embryo. This therapy offers a permanent effect for all who inherit the target gene. Successful therapies increases the possibility of eliminating certain diseases from a certain family, and ultimately from the entire population.
Advantages
Disadvantages
- gets rid of certain diseases

-gives patients new genes so they do not have the disease and so that the disease does not come back

-provides a way of treatment when the in the past, the disease would be incurable

-gives an advantage to someone who has been born with a genetic disorder

-replaces non-functional genes which may give someone a chance to live a normal life
-very costly, not consistent and fairly new

-hard to deliver genes efficiently in a uniform manner throughout a tissue system

-patients may have to undergo several treatment therapies

-there are several possible risks
Gene Therapy
Catholic Church's View
- ALL Catholic churches are against the scientific study of Genetic Therapy, or genetics used to enhance the human structure. However, the Church accepts few forms of gene therapy that are use to treat illnesses

-the Catholic churches accepts SOMATIC gene therapy

-the Catholic Church released a document in 2008 called Dignitas Personae. In it, the Church states, "a human is sacred from conception, and no form of experimenting on embryos or use of embryonic stem cells is at all acceptable in any form." Therefore, GERM LINE gene therapy is NOT accepted because of the potential risks that can affect the embryo
Effects on Genetic Diversity
- the use of gene therapy can decrease the genetic diversity of species, if no regulations are placed

- if no regulations are placed, people will use the opportunity to change their traits (e.g appearance, IQ)

- direct manipulation of our genes, enables us to control our own evolution, which may potentially decrease the genetic diversity of species
On September 14, 1990, a four-year old girl became the first gene therapy patient. The gene therapy was used to treat her adenosine deaminase (ADA) deficiency which is a genetic disease that leaves a person defenseless against infections.
- Gene therapy can target wrong cells. The vector is able to affect more than one type of cells, and not just the targeted cells containing mutated genes. If this happens, healthy cells may be damaged, causing other illnesses.
- Gene therapy raises the possibility of a tumor formation. If the new genes are inserted in the wrong location in the patient's DNA, there is a chance that the insertion might lead to tumor. This has occurred in the past in some clinical trials.
Risks
First Gene Therapy Trial
Social & Ethical Implications
Not much research has been put into gene therapy, which results in a lot of questions from the public. Here are a few questions that continuously appear when gene therapy is discussed:

-"Will high cost of gene therapy make it only available to the wealthy?"

-"Who decides which traits are normal and which constitute a disability or disorder?"

-"Could the widespread use of gene therapy make society less accepting of people who are different?"

-"Are we playing with God?"
The process:
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