Gene Therapy: Strategies and Challenges

Play an AI-generated podcast conversation about this lesson

In molecular biology, what are homologs?

Genes that produce different proteins in different species
Genes that have identical functions in different species (correct)
Genes that produce identical proteins in different species
Genes that have different functions in different species

What role do model organisms play in identifying disease genes?

They help identify tumor suppressor genes in humans
They can be used to manipulate human genetics for experimental purposes
They can be used to test gene therapy and drug-based therapeutic approaches for human diseases (correct)
They are not useful in identifying disease genes in humans

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

They can be manipulated in humans for experimental purposes
They play a crucial role in preventing the development of cancer and other diseases (correct)
They have been shown to be similar to human genes based on DNA sequence
They are not related to molecular biology's study of human disease conditions

What are tumor suppressor genes?

Genes that produce proteins to keep cancer formation in check (B) Signup and view all the answers

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 (D) Signup and view all the answers

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 (C) Signup and view all the answers

What is the purpose of Herceptin in breast cancer treatment?

To bind and inhibit the overexpressed HER-2 protein (B) Signup and view all the answers

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 (C) Signup and view all the answers

What is the main focus of gene therapy?

To deliver therapeutic genes into the human body to correct disease conditions created by faulty genes (C) Signup and view all the answers

How are monoclonal antibodies used in cancer treatment?

To target specific molecules found in cancer cells (D) Signup and view all the answers

What is the potential application of nanoparticles in medicine?

To unclog arteries and detect and eliminate cancer cells (D) Signup and view all the answers

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. (A) Signup and view all the answers

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

Adenovirus (D) Signup and view all the answers

How do viruses infect human cells?

Viruses bind to and enter cells, releasing genetic material into the nucleus or cytoplasm. (D) Signup and view all the answers

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. (A) Signup and view all the answers

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. (D) Signup and view all the answers

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

It is a pump protein that removes chloride ions from cell membranes. (A) Signup and view all the answers

Why is gene therapy for cystic fibrosis not yet dependable?

DNA delivered via liposomes does not integrate into chromosomes, requiring multiple reapplications. (B) Signup and view all the answers

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. (D) Signup and view all the answers

'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. (C) Signup and view all the answers

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

Efficiency of viruses at infecting many types of human cells. (B) Signup and view all the answers

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) (B) Signup and view all the answers

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. (D) Signup and view all the answers