Gene Therapy: Strategies and Challenges

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23 Questions

In molecular biology, what are homologs?

Genes that have identical functions in different species

What role do model organisms play in identifying disease genes?

They can be used to test gene therapy and drug-based therapeutic approaches for human diseases

How do tumor suppressor genes relate to molecular biology's study of human disease conditions?

They play a crucial role in preventing the development of cancer and other diseases

What are tumor suppressor genes?

Genes that produce proteins to keep cancer formation in check

How are researchers working on developing drugs related to tumor suppressor genes?

By designing drugs that bind to the protein products of tumor suppressor genes to inhibit or activate their function

What is the role of the genes BRCA1 and BRCA2 in relation to breast cancer?

They can be involved in breast cancer, and their specific involvement can be determined through genetic analysis

What is the purpose of Herceptin in breast cancer treatment?

To bind and inhibit the overexpressed HER-2 protein

How do artificial bloods differ from donated blood?

Artificial bloods are cell-free solutions that have molecules to carry and release oxygen, while donated blood contains red blood cells and plasma

What is the main focus of gene therapy?

To deliver therapeutic genes into the human body to correct disease conditions created by faulty genes

How are monoclonal antibodies used in cancer treatment?

To target specific molecules found in cancer cells

What is the potential application of nanoparticles in medicine?

To unclog arteries and detect and eliminate cancer cells

What is the primary difference between ex vivo gene therapy and in vivo gene therapy?

Ex vivo gene therapy involves removing cells from the patient, treating them, and then reintroducing them, while in vivo gene therapy involves directly introducing genes into tissues and organs in the body.

Which virus is commonly used as a vector for carrying therapeutic genes in gene therapy?

Adenovirus

How do viruses infect human cells?

Viruses bind to and enter cells, releasing genetic material into the nucleus or cytoplasm.

What is the primary function of small interfering RNAs (siRNAs) in RNA interference (RNAi)?

siRNAs serve as a complementary base pair to the mRNA, blocking it from translation.

What was the defect in the gene adenosine deaminase (ADA) that led to severe combined immunodeficiency (SCID)?

The defect led to the accumulation of toxic dATP, which was harmful to T cells.

Which of the following best describes cystic fibrosis transmembrane conductance regulator (CFTR)?

It is a pump protein that removes chloride ions from cell membranes.

Why is gene therapy for cystic fibrosis not yet dependable?

DNA delivered via liposomes does not integrate into chromosomes, requiring multiple reapplications.

What is one of the potential risks associated with using viruses as vectors for gene therapy?

'Death of Jesse Gelsinger' due to complications related to adenovirus vector in 1999.

'Can gene expression be controlled?' - This question primarily pertains to which aspect of gene therapy?

'Antisense RNA technology' as a way to block translation of mRNA molecules.

'Viruses Could Make Good Vectors' - This statement is primarily associated with:

Efficiency of viruses at infecting many types of human cells.

Which technology has been used successfully in cell culture but has yet to live up to its promise as a treatment for disease?

RNA interference (RNAi)

What was the outcome of scientist beginning gene therapy trials for cystic fibrosis after cloning the CFTR gene in 1989?

Gene therapy trials led to an increase in average life span for CF patients from 19 years to over 37 years.

Explore the primary strategies of gene therapy, including ex vivo and in vivo approaches, and the challenges of delivering genes only to intended tissues. Learn about the effectiveness and safety of gene therapy in sending genes to the proper tissues and organs.

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