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    Gene Therapy

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    api-320549212
    This document provides information about gene therapy and vectors. It includes questions about gene therapy candidates, delivery techniques, and vector types. Cystic fibrosis is identified as a good candidate for gene therapy because it meets the five criteria. The Adeno-Associated Virus is selected as the best vector for cystic fibrosis gene therapy because it does not cause immune responses and can infect both dividing and non-dividing cells. Challenges of gene therapy include targeting the correct cells and tissues, avoiding immune responses, and ensuring the vector does not disrupt other genes.

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    © All Rights Reserved
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    Gene Therapy

    Uploaded by

    api-320549212
    139 views3 pages
    This document provides information about gene therapy and vectors. It includes questions about gene therapy candidates, delivery techniques, and vector types. Cystic fibrosis is identified as a good candidate for gene therapy because it meets the five criteria. The Adeno-Associated Virus is selected as the best vector for cystic fibrosis gene therapy because it does not cause immune responses and can infect both dividing and non-dividing cells. Challenges of gene therapy include targeting the correct cells and tissues, avoiding immune responses, and ensuring the vector does not disrupt other genes.

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    This document provides information about gene therapy and vectors. It includes questions about gene therapy candidates, delivery techniques, and vector types. Cystic fibrosis is identified as a good candidate for gene therapy because it meets the five criteria. The Adeno-Associated Virus is selected as the best vector for cystic fibrosis gene therapy because it does not cause immune responses and can infect both dividing and non-dividing cells. Challenges of gene therapy include targeting the correct cells and tissues, avoiding immune responses, and ensuring the vector does not disrupt other genes.

    Copyright:

    © All Rights Reserved
    Download as DOCX, PDF, TXT or read online from Scribd
    Download as docx, pdf, or txt
    139 views3 pages

    Gene Therapy

    Uploaded by

    api-320549212
    This document provides information about gene therapy and vectors. It includes questions about gene therapy candidates, delivery techniques, and vector types. Cystic fibrosis is identified as a good candidate for gene therapy because it meets the five criteria. The Adeno-Associated Virus is selected as the best vector for cystic fibrosis gene therapy because it does not cause immune responses and can infect both dividing and non-dividing cells. Challenges of gene therapy include targeting the correct cells and tissues, avoiding immune responses, and ensuring the vector does not disrupt other genes.

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    You are on page 1of 3

    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.

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