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Gene Therapy

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Allie Rutz

on 12 March 2014

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Transcript of Gene Therapy

There are two specific types of gene therapy:
What is Gene Therapy?
Gene therapy
Gene Therapy
Germline Gene Therapy
Gene therapy targeting gametes (egg and sperm cells)
Gene is inserted into the gamete cell
This gene can be passed on genetically
Somatic Gene Therapy
Only somatic (body)
cells are targeted- not

Only effects individual treated

Cannot be genetically passed on to offspring
Main Sources:
Source 1:

Source 2:

Source 3:

Researchers are trying:
Replacing a mutated gene that causes disease with a healthy copy of the same gene.
By Allie Rutz
Source 1
Source 3

Source: http://images.businessweek.com/ss/09/06/0604_decade_of_disappointments/image/gene_therapy_getty.jpg
is an experimental technique that uses genes to treat or prevent diseases.
Source 1
Inactivating, or "knocking out" a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight the disease.
Source 1
Source: http://topnews.in/health/files/Gene-therapy_0.jpg
How does it work?
-Only test on diseases that have no cure

-A gene inserted directly doesn't usually work,
so gene therapy uses vectors.

- are carriers that are genetically engineered to deliver the gene.

-Vectors can be injected or pumped through an IV into a specific tissue
-Cells can be taken out of the patient, injected with vectors, and then placed back in the patient
-If the vector infection is successful, the cell uses the info in the gene to build RNA and protein molecules.
Source: Youtube and Discovery News
Source 1
Source 1
Sources 1 and 2
What kind of diseases could gene therapy help?
Source: http://2.bp.blogspot.com/_XNVS6Bkyaac/TBOSuPyUjzI/AAAAAAAAAzA/gXfIuiSLiWc/s1600/Gene_Therapy+cartoon.jpg
Genetic Diseases
Cystic Fibrosis
Sickle Cell Anemia
Muscular Dystrophy
Source: http://www.intechopen.com/source/html/43162/media/image1_w.jpg
Sources 1 and 3
What makes a good candidate?
Diseases with single gene mutations
Must know:
-Which specific genes to pursue
-Biology of the disorder

Must have access to the healthy gene

Must get rid of misbehaving gene before applying the healthy gene
Source: http://healthmap.files.wordpress.com/2011/01/human-gene-therapy.jpg
Source 2
Technical Challenges
-Better ways to deliver genes
-Better ways to target the genes
-Need to ensure genes can be controlled by the body
Source: http://static.ddmcdn.com/gif/gene-therapy-1.jpg
Source 1
Is gene therapy safe?
- Can possibly lead to serious health risks
-Risks are unpredictable because it's new
-FDA regulates gene therapy
-National Institutes of Health provide guidelines for investigations and institutions
Source 1
Ethical Issues
How can "good" and "bad" uses be distinguished?
Who decides which traits are normal and which constitute a disability or disorder?
Will the high costs of gene therapy make it available to only the wealthy?
Could the widespread use of gene therapy make society less accepting of people who are different?
Should people be able to use gene therapy to enhance basic human traits like height, intelligence, or athletic ability?
Source 1
Source: http://www.pjms.com.pk/issues/janmar09/fig_tab/20.article19_tab2.gif
Ethical Issues with Germline Gene Therapy
-Could affect development of fetus in unexpected ways
-Unborn children don't have a say in the treatment
-US government doesn't give federal funds to germline gene therapy
Source 1
Source: Youtube and Discovery News
Youtube and Discovery News
Viruses are most commonly used because:
-They can infect the cell while carrying the gene
-The virus can be modified so it won't spread disease
Vectors can be:
Source 1
Full transcript