Biotech Ppt Gene Therapy

8/19/2019 Biotech Ppt Gene Therapy

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Gene Therapy!By Marian Crockett


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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 thefunctioning gene, just like we learnedabout in class!

This idea has the potential to cure a lot of previously incurable genetic diseases


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Some Genetic Diseases

What diseases can Gene Therapy potentially cure?

X-linked severe combined immunodeficiency X-"#$%

Type # $iabetes

"ystic &ibrosis

"ancer

'arkinson(s $isease #nherited )lindness

*untington(s $isease

+nd more!


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What can you use as vectors?

ou can use many kinds of viruses as vectors, such as

.etroviruses, +denoviruses, +deno-associated viruses, or the

herpes simple viruses

0r as an alternative to viruses

ou can directly insert the $1+ into the cell with limited

success%

+rtificial lipid spheres can be created to carry the $1+ to transfer

to the cells


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How to use Vectors

#nsert the .1+ version of the normal allele intothe retrovirus vector%

2et the retrovirus infect bone marrow cells thathave been removed from the patient

#nsert the virus( $1+ with the normal alleleinside% into the chromosome of the cell of the

patient

2et the cells grow in culture to make sure they allhave the chromosome

#nject the cells into the patient, they should allnow carry the new chromosome!!


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Ethica uestions"

3ill tampering with human genes lead to eugenics a

deliberate effort to control the genetic makeup of humans%4

#s this tampering with evolution4 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 now4

hould we be changing the genetic information of embryos or

is that considered genetic engineering4

What do you think???


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Sti in #ro$ress

3hile Gene Therapy sounds great as an idea, it(s still

being researched on thoroughly and scientists don(t know

everything about it5 #t(s a work in progress5

There are still 6uestions

*ow can you control the gene to make the rightamount of substance at the right time at the right place4

*ow can we make it so the insertion of the gene

doesn(t harm some other important cell function4


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%ecent Deveopments

+s 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%5 The lack of hairs caused the deafness5

7sing mice, scientists have inserted 8 tumor suppressing genes

that have significantly reduced the si9e and number of lung

cancer tumors5 This is helpful for curing lung cancer in

humans5


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More %ecent Deveopments

1ew tests have shown that gene therapy shows improvement in

sight for a type of inherited blindness called 2eber:s congenital

amaurosis!

+ 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 inserted5


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Works Cite&

.ossi, 25 8;;


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Works Cite&

8;>>, +ugust 8;, 8;>8 fromhttp//www5ornl5gov/sci/techresources/*uman?Genome/medicine/gene

therapy5shtml

*echt, &5 8;;%5 Gene Therapy- The &uture is *ere!

MedicineNet.com. .etrieved =ay >;, 8;>8 fromhttp//www5medicinenet5com/script/main/art5asp4articlekeyB>8CC8

$augherty, D5 8;;@%5 Biotechnology. aint 'aul 'aradigm 'ublishing,

#nc


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Works Cite&

Gene Therapy5 Gene School. .etrieved =ay >;, 8;>8 from

http//library5think6uest5org/8EAFF/gene?therapy5htm

"otrim, + and )ruce )aum5 Gene Therapy ome *istory,+pplications, 'roblems, and 'rospects5 Sage ournal!. .etrieved =ay >;, 8;>8 from

http//tp5sagepub5com/content/C/>/F@5full

"ampbell, 1eil +5 and Hane ) .eece5 8;;A%5 Biology5 an&rancisco 'earson )enjamin "ummings


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Works Cite& 'pictures(

https//youngbloodbiology5wikispaces5com/file/view/genether

apy85jpg/C8/;/

$iabetes5jpg

http//upload5wikimedia5org/wikipedia/commons/;/;b/+denovi

rus5jpg

http//upload5wikimedia5org/wikipedia/commons/thumb/A/A>/"

&treatmentvest85H'G/8;p-"&treatmentvest85H'G
https://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpghttps://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpghttps://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpghttp://societynews.org/wp-content/uploads/2012/03/Diabetes.jpghttp://societynews.org/wp-content/uploads/2012/03/Diabetes.jpghttp://societynews.org/wp-content/uploads/2012/03/Diabetes.jpghttp://societynews.org/wp-content/uploads/2012/03/Diabetes.jpghttps://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpghttps://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpghttps://youngbloodbiology.wikispaces.com/file/view/genetherapy2.jpg/64968388/genetherapy2.jpg

Biotech Ppt Gene Therapy

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