L5. Gene therapy II - AI MCQs

<p>Chronic coughing and shortness of breath. (C)</p> Signup and view all the answers

<p>Air pollution and chemical fumes contribute to its development. (A)</p> Signup and view all the answers

<p>B cells are primarily responsible for attacking invading bacteria, viruses, and toxins. (D)</p> Signup and view all the answers

<p>It leads to an accumulation of dATP that inhibits DNA synthesis. (B)</p> Signup and view all the answers

<p>It entails altering cells outside the body before reintroducing them. (D)</p> Signup and view all the answers

<p>The correct CFTR gene enables cells to produce normal CFTR proteins. (B)</p> Signup and view all the answers

<p>NK cells (B)</p> Signup and view all the answers

<p>It prevents any disruption to the existing genome. (D)</p> Signup and view all the answers

<p>It represents a severe compromise in the immune system. (B)</p> Signup and view all the answers

<p>Gene therapy for SCID does not involve modifying the patient's immune cells. (C)</p> Signup and view all the answers

<p>T cells can completely replicate and function normally. (D)</p> Signup and view all the answers

<p>Both arms of the adaptive immune system are significantly impaired in SCID. (B)</p> Signup and view all the answers

<p>To enable cells to synthesize functional CFTR proteins. (A)</p> Signup and view all the answers

<p>It supports the normal functioning of lymphocyte development. (B)</p> Signup and view all the answers

<p>Non-integrating gene therapy relies on stable integration. (A)</p> Signup and view all the answers

<p>This therapy often involves the delivery of corrected CFTR genes to epithelial cells. (C)</p> Signup and view all the answers

<p>Gene therapy can be used to treat a wide range of genetic disorders. (A)</p> Signup and view all the answers

<p>Normal levels of functional T cells. (A)</p> Signup and view all the answers

<p>Successful integration into the genome can be inconsistent. (A)</p> Signup and view all the answers

<p>Enzyme replacement therapy has been shown to be beneficial for patients. (C)</p> Signup and view all the answers

<p>It results in impaired development of immune cells. (D)</p> Signup and view all the answers

<p>Administering hydroxyurea as a primary treatment. (A)</p> Signup and view all the answers

<p>B cells act by physically phagocytizing pathogens. (D)</p> Signup and view all the answers

<p>Targeting lung tissues for gene delivery remains a significant hurdle. (A)</p> Signup and view all the answers

<p>Blood transfusion as a means to deliver genetic material. (D)</p> Signup and view all the answers

<p>This therapy has been shown to be ineffective in young children. (B)</p> Signup and view all the answers

<p>It is guaranteed that gene therapy will provide a permanent cure for all patients. (A)</p> Signup and view all the answers

<p>The risk of immune reactions against viral vectors. (C)</p> Signup and view all the answers

<p>Regularly altering the genetic material being introduced. (B)</p> Signup and view all the answers

<p>Oncolytic agents are genetically engineered viruses that specifically target cancer cells. (B)</p> Signup and view all the answers

<p>It is the most commonly used viral vector for gene transfer in clinical trials. (D)</p> Signup and view all the answers

<p>Gene transfer techniques solely rely on bacterial vectors. (C)</p> Signup and view all the answers

<p>Access to gene therapy might not be equitable. (A)</p> Signup and view all the answers

<p>Herpes simplex virus type I is ineffective against cancer cells. (D)</p> Signup and view all the answers

<p>Enhancement of cancer cell metabolism (A)</p> Signup and view all the answers

<p>The long-term effects of retroviral vectors are well-studied and fully understood. (B)</p> Signup and view all the answers

<p>They cause cellular death upon expression. (B)</p> Signup and view all the answers

<p>Cost-effectiveness in production. (A)</p> Signup and view all the answers

<p>The ability to genetically enhance non-cancerous individuals. (B)</p> Signup and view all the answers

Various methods to correct the defective gene causing Sickle Cell Anemia, including gene editing and gene replacement.

A genetic disorder in which the immune system is very weak or non-functional.

A gene therapy method where the new gene is not incorporated into the existing DNA.

A gene therapy method where the new gene is integrated into the host's DNA.

A gene that encodes for the cystic fibrosis transmembrane conductance regulator protein, crucial for regulating salt and water balance in cells.

A genetic disorder caused by a mutation in the CFTR gene, leading to thick mucus buildup in the lungs and other organs.

A disease caused by a mutation in a single gene.

A gene therapy approach where cells are removed from the body, genetically modified, and then returned.

Gene therapy approaches for treating cystic fibrosis.

A disorder causing lung damage.

Using gene therapy to treat single-gene disorders.

Gene therapy methods for treating severe combined immunodeficiency.

Gene therapy approaches for treating sickle cell anemia.

A lung disease, often caused by alpha-1 antitrypsin deficiency.

Explain various approaches to treat certain diseases using genes.

Explaining gene therapy strategies for treating cystic fibrosis.

Explaining how gene therapy could potentially treat this deficiency.

Describing gene therapies to treat severe combined immunodeficiency.

The new copy of the gene could be inserted into a part of the genome that contains critical information, potentially disrupting normal gene function.

The therapeutic gene is not permanently integrated into the host's DNA, so the effect is temporary and the treatment may need to be repeated.

The adenovirus used for gene delivery may trigger an immune response, potentially limiting the effectiveness of the therapy.

Emphysema is primarily caused by smoking, but air pollution, chemical fumes, and long-term exposure to irritants can also contribute to its development.

Emphysema symptoms include shortness of breath, coughing, and fatigue.

A gene therapy approach using viruses that have been genetically engineered to target and kill cancer cells. These viruses replicate inside the cancer cells, leading to cell death.

Oncolytic vectors kill cancer cells through several mechanisms: 1) Viral replication and spread: They reproduce inside the cancer cells and burst them open, releasing more viruses. 2) Expression of cytotoxic proteins: They deliver genes that produce toxic substances specifically targeting cancer cells. 3) Cell lysis: They cause the direct destruction of the cancer cell.

Different viruses are used as vectors in oncolytic therapy, each with advantages and disadvantages. Some examples include adenovirus, herpes simplex virus type I, and reovirus.

This involves directly introducing a gene into cancer cells or surrounding tissues. This gene could be a suicide gene causing cell death or an antiangiogenesis gene preventing blood vessel growth that feeds the tumor.

These are genes that are introduced into cancer cells and cause cell death when expressed. They act like a 'kill switch' within the cell.

These genes are introduced to block the formation of new blood vessels that supply nutrients to the growing tumor, starving it.

Concerns arise about the potential consequences of altering the genetic makeup of future generations through germline therapy.

One potential long-term risk of using retroviral vectors is the possibility of inducing cancer.

A major ethical consideration is ensuring fair and equitable access to gene therapy, which can be costly and complex.

A key ethical concern is the potential for unforeseen negative consequences of introducing new genes into the human genome.

A rare genetic condition where the body's immune system is severely compromised due to faulty T-cell and B-cell development.

White blood cells crucial for adaptive immunity, responsible for recognizing and attacking specific pathogens. There are two main types: B cells and T cells.

Lymphocytes that produce antibodies, proteins that bind to specific antigens on pathogens to neutralize them.

Lymphocytes that directly attack infected cells or cancer cells, or regulate the immune response.

Adenosine deaminase, an enzyme critical for breaking down purines, a type of building block for DNA.

A nucleotide that can build up when ADA is defective, inhibiting DNA synthesis.

An enzyme crucial for converting ribonucleotides to deoxyribonucleotides, necessary for DNA synthesis.

A regulatory mechanism where the product of a pathway inhibits an earlier step in that pathway.

One of the two types of nitrogenous bases found in DNA and RNA, the others being pyrimidines.

ADA deficiency leads to a buildup of dATP, which inhibits DNA synthesis, particularly affecting the development of T cells and B cells, leading to a severely compromised immune system.