Exam 31: Gene Therapy

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.

Gene therapy strives to _______.

Gene therapy is_______ .

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?

Which gene therapy is currently approved in the United States?

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

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_______ .

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 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.

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?

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.

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

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 serious concern about gene therapies and their commercialization is_______

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.

Lentiviral vectors are preferred over adenoviral vectors because_______ .

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?

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

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.

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