Deck 31: Gene Therapy

Question 1

A serious concern about gene therapies and their commercialization is_______

A)their cost to the consumer
B)their speedy development process
C)the ease of achieving regulatory approval
D)the lack of individuals treatable with the drug

Answer 1

their cost to the consumer

Question 2

Which gene therapy is currently approved in the United States?

A)enhancement gene therapy
B)somatic gene therapy
C)stem cell gene therapy
D)germ- line gene therapy

Answer 2

B

Question 3

Treatment of ADA- SCID is often considered the first success for gene therapy.Ashanti DeSilva is a young girl that suffered from an autosomal form of SCID and was treated using retroviral gene therapy.Explain why the treatment is not an undisputed success.

Answer 3

While Ashanti's immune system was bolstered by the gene therapy,she also received regular injections of adenosine deaminase enzyme.This made it unclear whether the adenosine deaminase found in her system was due to the active gene delivered by the gene therapy or the injections of purified adenosine deaminase.

Question 4

Despite the promise of gene therapy treating disease,there were two major setbacks to clinical trials.This setbacks brought to light the disadvantages of using retroviral vectors for gene therapy,which include_______ .

A)the unknown response of a patient to the retroviral vector
B)controlled insertion of the therapeutic gene in the host genome
C)certain viral vectors do not insert in important genes of the host
D)the viral vector will not impact the host

Question 5

ZFNs and TALENs have high specificity for their target cleavage site.How is this specificity achieved?

Question 6

The use of CRISPR- cas has revolutionized the gene therapy field.One of the first uses of CRISPR- cas to edit genes in vivo was done at MIT.What disease did they cure?

A)B- thalassemia
B)RDEB
C)type 1 tyrosinemia
D)HIV

Question 7

The gene PD- 1 plays an important role in immune response to tumor cells.How would using gene editing to knock out this gene help T cells recognize and kill cancer cells?

A)PD- 1 will be expressed in the cytosol of the T cell.This protects PD- 1 from cancer cells.
B)PD- 1 will be expressed in the cytosol of the cancer cell.This protects PD- 1 from the T cell.
C)PD- 1 will not be expressed on the cancer cell surface.This prevents T cell recognition.
D)PD- 1 will not be expressed on the T cell surface.This circumvents one line of the cancer cells defense.

Question 8

Gene therapy to treat Leber congenital amaurosis has recently shown some promise in revitalizing the gene therapy field.This is due in large part to injecting adeno- associated virus loaded with RPE65 just under the retina.What does the product of gene RPE65 do?

A)metabolizes retinal
B)metabolizes cone cell waste
C)metabolizes retinol
D)metabolizes rod cell waste

Question 9

CTL019 was the first gene edited CAR T cell therapy approved by the FDA.Initial treatments of patients with CTL019 demonstrated over _______remission rate after the first treatment.Exemplifying the power of gene editing in treating acute lymphoblastic leukemia.

A)95%
B)90%
C)99%
D)80%

Question 10

What is the target of the first CRISPR- cas gene editing in humans?

A)COL7A1
B)CAR T cells
C)FUH
D)PD- 1

Question 11

Hematopoietic stem cells HSCs are particularly useful in gene therapy because .

A)they differentiate into all tissue types
B)they are useable across many patients without rejection fears
C)they are easy to obtain from patients
D)they are short lived in vitro

Question 12

With gene therapy being at the cutting edge of medical technologies,it is not surprising that serendipitous treatments are discovered.Explain how treating an HIV- positive patient with stem cells from an HIV- resistant donor cured him or her of HIV while targeting his or her acute myeloid leukemia.

Question 13

Minimizing off- target effects of gene therapy is a concern with _ _.

A)in vivo gene therapy
B)the use of adenovirus therapy
C)autologous gene therapy
D)ex vivo gene therapy

Question 14

Gene therapy is_______ .

A)able to treat any type of cell no matter where it is in the body
B)not a good candidate for cancer therapeutics
C)an example of translational medicine
D)capable of treating diseases associated with an unknown gene

Question 15

Lentiviral vectors are preferred over adenoviral vectors because_______ .

A)the lentivirus cannot infect nondividing cells
B)they do not have to be engineered by taking out their replicative genes
C)the inserted DNA forms episomes,which prevent insertion mutations
D)the inserted DNA cannot be reproduced when cells divide

Question 16

Gene therapy strives to _______.

A)mitigate the symptoms of a disease state
B)provide new gene sequences to make people stronger
C)cure the disease state
D)cut out defective genes

Question 17

A patient experiencing a disease state due to a gene deletion was treated with adenovirus gene therapy.The gene therapy was successful and the gene deletion was corrected.However,the patient began to develop cancer not long after the gene therapy.Develop a theory as to why the cancer presented so rapidly after gene therapy.

Question 18

Viral vectors are not the only method being studied for gene therapy purposes.Which of the following is a nonviral delivery method for gene therapy?

A)DNA bound to the surface of liposomes
B)DNA covered with protein
C)electrically stimulating cells to take up DNA
D)gene pills

Question 19

One gene therapy that does not rely on gene editing is RNA interference.This therapy uses _______to interfere with expression of proteins at the translational level.

A)mRNA
B)miRNA
C)mtRNA
D)tRNA

Question 20

In 2015,a group of Chinese researchers used CRISPR- cas gene editing to edit the HBB gene in 86 human embryos.What were the conclusions form this study?

A)71 of the 86 embryos contained the desired gene edit.
B)CRISPR- cas is safe for use in human patients.
C)CRISPR- cas gene editing had no negative effects on the surviving embryos.
D)CRISPR- cas was not ready for use in human patients.