MI Unit 2.2 Gene Therapy

What is gene therapy ?

• Pre-clinical testing for inherited diseases in newborns

• Treatment of diseases caused by genetic defect

• Genetic engineering using recombinant DNA technology

• Cancer treatment using in vitro cultured stem cells

Gene therapy can be administered to people who suffer from all these disorders EXCEPT for

• Down Syndrome

• Cystic Fibrosis

• Sick Cell Disease

• Hemophilia

Gene therapy can only be administered to patients using viral vectors

• True

• False

Which of the following is a preferred vector for transferring genes to nerve cells?

• Adeno-associated viral vector

• Herpes Simplex viral vector

• Retroviral vector

• Vaccinia vector

The effectiveness of a gene therapy trial can be influenced by (check all that apply)

• The receptors on the viral vectors

• The genome holding capacity

• The age of the patient

• The severity of the disorder symptoms

Which of the following is not a risk or challenge of gene therapy?

• Can be passed on to offspring

• Could cause cancer

• Short term effects

• Protein my not transcribed and translated in the targeted tissue

A nasal spray containing adenovirus carrying a functional human CFTR gene is used to treat cystic fibrosis is an example of which type of gene therapy?

• In situ

• In vivo

• Ex vivo

• Vaccine

In gene therapy, in order to be successful, the healthy gene inserted into a target cell must...

• Take over and kill the defective gene

• Be inserted manually into the cell's mitochondria

• Become attached to the cell's mRNA molecules

• Be able to make the correct amount of the protein needed

A three-year-old boy was being treated for SCID using a modified retrovirus, but the boy developed a leukemia-like condition. This is a type of caner. What appeared to have caused this?

• The disorder is know to cause leukemia in most patients

• The retrovirus inserted near another gene

• The virus was not properly altered to not cause disease

• The virus caused an immune response

The ABDS marketing department has identified Oma, Inc. as a biotech company that is developing gene therapies.Oma Inc. would like to target non-small cell lung cancer (75% of all lung cancers) with gene therapy. They need a vector to introduce p53, a tumor suppressor gene (2,150 base pairs long), directly into the tumor. The vector must target the lung cells directly and the potential immune response must be considered.

• Retrovirus

• Adenovirus

• Adeno-Associated Virus

• Herpes Simplex Virus

• Not a good gene therapy candidate