Name:RaneDutcher

Logonto:http://gslc.genetics.utah.edu/units/genetherapyandexplorethismoduletofindthe
answerstothequestionsbelow.
Hint:theSearchfeatureonthiswebsitemayormaynothelpyoufindwhatyouarelookingfor;itis
besttogothroughthemoduletofindtheanswers.
Questions:
1.Whatisgenetherapy.Atransplantationofgenesintothecellsofahostthathasmissingordefective
genes.
2.Whatarethefivecriteriaforagenetherapycandidatedisease?Conditionresultsfrommutationsinone
ormoregenes.Mustknowwhichgenesareinvolved.Mustknowthebiologyofthedisorder.Addinga
normalgenehastoaffecttheproblems.Mustbepossibletogetthegenetotheinfectedcells.
3.CaseStudy:Iscysticfibrosisagoodcandidateforgenetherapy?Whyorwhynot?
Yes,cysticfibrosisisagoodcandidateforgenetherapybecauseitmeetsthefivecriteria.
4.Whatarethehallmarksofsuccessfulgenedeliveryingenetherapytrials?
Gettingtherightcells,activatingthegene,combiningthegenewiththecell,avoidanyharmfulside
effects.
5.Explaintheinvivotechniqueofdeliveringgenestoapatientscells.
Theinvivotechniqueiswherethevectorthatcontainsthegeneisinjectedusinganeedledirectlyintothe
hostsbody.
6.Explaintheexvivotechniqueofdeliveringgenestoapatientscells.
Infectedcellsareisolatedandtakenoutoftheinfectedtissueofthehost,thenplacedontoapetridish.The
vectorwiththegeneisintroducedtothecells,wheretheprocesstakesplace.
7.Whatarevectors?WhatarethesixdifferenttypesofvectorscontainedintheVectorToolbox?
Avectoris:Somethingthatgoesintothebodythathasahelpfulgeneinsertedinsideit.

Thesixdifferenttypesofvectorsare:Retrovirus,Adenovirus,AdenoAssossiatedVirus,Herpes
SimplexVirus,Liposome,NakedDNA

8.GothroughtheVectorToolbox.FillouttheattachedVectorTypesTableattheendofthisdocument.
9.Whichvectorwouldbethebestchoiceforacysticfibrosisgenetherapy?
Why?
TheAdenoAssociatedVirusisthebestchoiceforcysticfibrosisbecauseitdoesnotcausean
immuneresponse,itlocatestherighttissue,anditcaninfectbothdividingandnondividingcells.

10.BecometheSpaceDoctoranddesignagenetherapyforoneofthethreegivenpatients.Filloutthe
following:
PatientDiseaseTissuetargeted
Tungs
Vectorchosen(consultthetoolboxifyouneedarefresher)
AdenoAssossiatedVirus
Wasyourtherapyinvivoorexvivo?
Invivo
Wasyourtherapysuccessful?Whyorwhynot?
Itwassuccessfulbecausetheviruscouldholdthegene,getintothecell,andthegeneintegratedto
anondisruptivelocationonchromosome19.
Listallofthevectorsthatworkedforthisgenetherapy
Adenovirus
AdenoAssociatedVirus
11.Whatarethechallengesofgenetherapy?Listandbrieflydescribeeachone:
a.Thevectorneedstogoaftertherightsystem;theherpesvirusgoesafterthenervoussystem.
b.Thevectorneedstocorrespondtothecellsdividinghabits;iftheydontthentheycangivethe
genetothecells.
c.Thevirusescanttriggeranimmuneresponse,orthevectorwouldjustgetcanceledout.
12.Whataresomeissuesinvolvedingenetherapy?

Not all vectors work all the time.

You need to know if it should be in vivo or ex vivo.
The virus remains in the body after therapy.

Retrovir
us

Adenovir
us

Herpes
Simple
x Virus
Double
Strand
ed DNA

Liposo
me

Naked
DNA

Double
Stranded
DNA

AdenoAssociat
ed Virus
Single
Strande
d DNA

How the
vector
carries
genetic
material
Maximum
lengthofDNA
thatcanbe
insertedinto
vector

Carries
Material in
Form of
RNA

Plasmid
s

Plasmi
d

8,000
base
pairs

7,500
base
pairs

5,000
Base
pairs

20,000
base
pairs

Not
Viral

Not
Viral

Advantages

Contains
enzymes
to
convert
RNA to
DNA

Infects
dividing
and non
dividing
cells

Will not
cause an
immune
respons
e

Does
not
disrupt
other
genes
in host
cell

Excelle
nt for
ex vivo
gene
therapy

Disadvanta
ges

Could
integrat
e to a
place
that
disrupts
another
gene

Will not
integrate
to a host
cells
genome

Needs a
helper
virus to
duplicat
e

Causes
an
immun
e
respon
se

Can be
toxic
Cant
integrat
e

Not
Toxic
and
does
not
genera
te
immun
e
respon
se
Will not
integra
te to
host
cells
genom
e.