Quiz 2_Gene therapy

What is one of the main purposes of gene therapy

for beta-thalassemia, as mentioned in the handout?

• To prevent viral infections structurally through genetic modification.

• To delete the mutated gene to stop production of a disease-causing protein.

• To boost the immune system.

• To enhance memory and learning capabilities.

What is the primary role of enhancers in transcription regulation,

according to the text in the handout?

• To turn transcription off by condensing chromatin

• To silence genes by binding repressors

• To turn transcription on from far distances or in multiple places

• To serve as promoter proximal elements

What is the advantage of using non-integrating Adeno-associated virus (AAV) in gene therapy?

• Integrates efficiently into ex vivo cells

• Targets cancer cells specifically as an oncolytic virus

• Forms an RNA virus that integrates into the genome

• Viral DNA becomes a circular episome

What is the primary cause of complications in thalassemia?

• Imbalance of alpha- and beta-globin chains.

• Excessive synthesis of beta-globin chains.

• Denaturation of hemoglobin.

• Lack of hematopoietic stem cells.

During erythropoiesis, which step marks the beginning of hemoglobin synthesis?

• Mature (specialized) cells

• Hematopoietic stem cells

• Immature (blast) cells

• Progenitor (precursor) cells

What method is used for stem cell mobilization in the context provided?

• Granulocyte colony-stimulating factor and plerixafor

• Blood transfusion

• Chemotherapy with busulfan

• Bone marrow aspiration

What is a key step to make space in the bone marrow

for donor hematopoietic stem cell engraftment?

• Chemotherapy for a few days.

• Intravenous infusion of stem cells.

• Expansion of CD34+ cells in vitro.

• Mobilization with granulocyte colony-stimulating factor.

Which of the following is an example of a regulatory transcription factor

involved in turning off transcription?

• General transcription factor

• Activator

• Repressor

• Coactivator

What is the main purpose of ex vivo gene therapy as described in the text?

• Using CRISPR technology to edit the genome

• Transfecting stem cells with proteins to activate them

• Suppressing immune reactions to transgene products

• Restoring normal function by correcting the mutated gene

What is the main purpose of in vivo gene therapy as described in the text?

• Correcting the mutated gene to restore normal function

• Enhancing the immune system to fight diseases

• Removing harmful bacteria from the body

• Inserting the missing gene to produce a needed gene