Gene Therapy!

By Marian Crockett

What is Gene Therapy?

Gene Therapy corrects genetic

disorders/defects/diseases by replacing
the defective gene with a normal one

Gene Therapy is uses vectors to insert the

functioning gene, just like we learned

about in class!

This idea has the potential to cure a lot of

previously incurable genetic diseases

Some Genetic Diseases

What diseases can Gene Therapy potentially cure?

X-linked severe combined immunodeficiency (X-SCID)
Type I Diabetes
Cystic Fibrosis
Cancer
Parkinsons Disease
Inherited Blindness
Huntingtons Disease
And more!

What can you use as vectors?

You can use many kinds of viruses as vectors, such as:

Retroviruses, Adenoviruses, Adeno-associated viruses, or the

herpes simplex viruses

Or as an alternative to viruses:

You can directly insert the DNA into the cell (with limited
success)

Artificial lipid spheres can be created to carry the DNA to transfer

to the cells

How to use Vectors

Insert the RNA version of the normal allele into

the retrovirus (vector)

Let the retrovirus infect bone marrow cells that

have been removed from the patient

Insert the virus DNA (with the normal allele

inside) into the chromosome of the cell of the
patient

Let the cells grow in culture to make sure they all

have the chromosome

Inject the cells into the patient, they should all

now carry the new chromosome!!

Ethical Questions

Will tampering with human genes lead to eugenics (a

deliberate effort to control the genetic makeup of humans)?

Is this tampering with evolution? Genetic variation is needed

in the gene pool, are we willing to risk the survival of our

species in the future by changing the genome now?
Should we be changing the genetic information of embryos or

is that considered genetic engineering?

What do you think???

Still in Progress

While Gene Therapy sounds great as an idea, its still

being researched on thoroughly and scientists dont know

everything about it. Its a work in progress.
There are still questions:
How can you control the gene to make the right

amount of substance at the right time at the right place?

How can we make it so the insertion of the gene
doesnt harm some other important cell function?

Recent Developments

As weird as it sounds, scientists have gotten gene therapy to

cure deafness in guinea pigs by inserting a gene that regrew

hair cells in their ears (that were destroyed by the
nonfunctioning gene). The lack of hairs caused the deafness.
Using mice, scientists have inserted 2 tumor suppressing genes

that have significantly reduced the size and number of lung

cancer tumors. This is helpful for curing lung cancer in
humans.

More Recent Developments

New tests have shown that gene therapy shows improvement in

sight for a type of inherited blindness called Leber's congenital

amaurosis!
A combination of using stem cells and gene therapy could lead

to a breakthrough new way to treat cystic fibrosis by making

the adult stem cells differentiate into airway epithelial cells that
have the turned-off cystic fibrosis gene inserted.

Works Cited

Rossi, L. (2004, December 20). Combined stem cell-gene

therapy approach seen as potential treatment for cystic fibrosis.

EurekAlert. Retrieved May 10, 2012 from
http://www.eurekalert.org/pub_releases/2004-12/uopmcsc121704.php
(2005, November 25). Gene Therapy. Health Canada.

Retrieved May 10, 2012 from http://www.hc-sc.gc.ca/srsr/biotech/about-apropos/gen_therap-eng.php

Works Cited

(2011, August 24). Gene Therapy. Human Genome Project

Information. Retrieved May 10, 2012 from

http://www.ornl.gov/sci/techresources/Human_Genome/medicine/gene
therapy.shtml
Hecht, F. (2004, September 21). Gene Therapy- The Future is Here!

MedicineNet.com. Retrieved May 10, 2012 from

http://www.medicinenet.com/script/main/art.asp?articlekey=12662
Daugherty, E. (2007). Biotechnology. Saint Paul: Paradigm Publishing,

Inc

Works Cited

Gene Therapy. Gene School. Retrieved May 10, 2012 from

http://library.thinkquest.org/28599/gene_therapy.htm

Cotrim, A and Bruce Baum. Gene Therapy: Some History,

Applications, Problems, and Prospects. Sage Journals.

Retrieved May 10, 2012 from
http://tpx.sagepub.com/content/36/1/97.full

Campbell, Neil A. and Jane B Reece. (2005). Biology. San

Francisco: Pearson Benjamin Cummings

Works Cited (pictures)

https://youngbloodbiology.wikispaces.com/file/view/genether

apy2.jpg/64968388/genetherapy2.
jpg
http://societynews.org/wp-content/uploads/2012/03/

Diabetes.jpg
http://upload.wikimedia.org/wikipedia/commons/0/0b/Adenovi

rus.jpg
http://upload.wikimedia.org/wikipedia/commons/thumb/5/51/C

Ftreatmentvest2.JPG/230px-CFtreatmentvest2.JPG