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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.jpgRecommended