Which of the following statements about viral vectors for gene therapy are correct?
A. In order to replicate, viruses introduce their genetic material into the host cell, tricking the host’s cellular machinery into using it as blueprints for viral proteins.
B. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell.
C. A number of viruses have been used for human gene therapy, including retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus (AAV).
D. Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host's genome, becoming a permanent part of the host's DNA in infected cells.
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Whichofthefollowingstatementsabout non-viralvectorsforgenetherapyarecorrect?
A. Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity.
B. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy.
C. Later technology remedied this deficiency.
D. Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.
Which of the following viruses are commonly used as vectors in gene therapy?
A. adenovirus
B. AAV
C. retrovirus
D. vaccinia virus
Whichofthefollowingvirusesarecommonlyusedasvectorsingenetherapy?
A. adenovirus
B. poxvirus
C. Herpes Simplex virus
D. lentivirus
Which of thefollowingdiseases havebeentestedforgenetherapyinpatients?
A. X-linkedseverecombinedimmunedeficiency(X-SCID)
B. SCIDlinkedwithdeficiencyofadenosinedeaminase(ADA-SCID)
C. lipoproteinlipasedeficiency(LPLD)
D. Leber'scongenitalamaurosis(LCA)
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