L5. Gene therapy II

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Questions and Answers

Which of the following is true regarding integrating gene therapy for Cystic Fibrosis?

The therapy is immediate and does not require further monitoring.

Integrating gene therapy does not carry any risks of genomic disruption.

The new CFTR gene copy is permanently integrated into the genome. (correct)

It requires repeated treatments for lasting effects.

Which of the following statements about adenovirus use in CF gene therapy is incorrect?

Lack of adenovirus receptors can lead to low transduction efficiency.

Immunogenicity is a challenge associated with adenovirus applications.

Adenovirus can effectively integrate into the host cell genome. (correct)

Adenovirus remains as an episome rather than integrating permanently.

Which of the following represents a disadvantage of non-integrating gene therapy methods?

They involve fewer treatment sessions compared to integrating methods.

Results are permanent and do not require monitoring.

The effects may diminish over time without repeated treatments. (correct)

They may provoke stronger immune responses.

Which of the following is a symptom associated with emphysema?

Chronic coughing and shortness of breath. Signup and view all the answers

Which of the following is an accurate statement regarding the causes of emphysema?

Air pollution and chemical fumes contribute to its development. Signup and view all the answers

Which statement about the role of B cells is correct?

B cells are primarily responsible for attacking invading bacteria, viruses, and toxins. Signup and view all the answers

Which of the following is an implication of the ADA deficiency related to SCID?

It leads to an accumulation of dATP that inhibits DNA synthesis. Signup and view all the answers

Which statement about ex vivo gene therapy is correct?

It entails altering cells outside the body before reintroducing them. Signup and view all the answers

Which of the following accurately describes the role of the CFTR gene in gene therapy for cystic fibrosis?

The correct CFTR gene enables cells to produce normal CFTR proteins. Signup and view all the answers

Which type of lymphocyte is NOT directly affected by SCID?

NK cells Signup and view all the answers

Which of the following is a characteristic of non-integrating gene therapy for cystic fibrosis?

It prevents any disruption to the existing genome. Signup and view all the answers

Which of the following correctly describes SCID?

It represents a severe compromise in the immune system. Signup and view all the answers

In the context of SCID, which assertion is incorrect?

Gene therapy for SCID does not involve modifying the patient's immune cells. Signup and view all the answers

Which of the following is an incorrect assumption about T cells in SCID?

T cells can completely replicate and function normally. Signup and view all the answers

Which of the following accurately reflects the relationship between SCID and lymphocyte function?

Both arms of the adaptive immune system are significantly impaired in SCID. Signup and view all the answers

Which option correctly summarizes the purpose of introducing a correct CFTR gene copy in cystic fibrosis treatment?

To enable cells to synthesize functional CFTR proteins. Signup and view all the answers

Which statement regarding the effects of dATP accumulation is incorrect?

It supports the normal functioning of lymphocyte development. Signup and view all the answers

Which statement about gene therapy approaches for cystic fibrosis is incorrect?

Non-integrating gene therapy relies on stable integration. Signup and view all the answers

Which of the following statements accurately describes a characteristic of gene therapy for cystic fibrosis?

This therapy often involves the delivery of corrected CFTR genes to epithelial cells. Signup and view all the answers

Which of the following statements about the gene therapy process is true?

Gene therapy can be used to treat a wide range of genetic disorders. Signup and view all the answers

Which of the following is NOT a characteristic of SCID?

Normal levels of functional T cells. Signup and view all the answers

Which statement about the limitations of gene therapy is accurate?

Successful integration into the genome can be inconsistent. Signup and view all the answers

Which statement is correct regarding the treatment of alpha-1 antitrypsin deficiency?

Enzyme replacement therapy has been shown to be beneficial for patients. Signup and view all the answers

Which of the following correctly describes a consequence of the genetic mutations causing SCID?

It results in impaired development of immune cells. Signup and view all the answers

Which of the following approaches is NOT associated with gene therapy for sickle cell anemia?

Administering hydroxyurea as a primary treatment. Signup and view all the answers

Which option wrongly describes the involvement of lymphocytes in immunity?

B cells act by physically phagocytizing pathogens. Signup and view all the answers

Which of the following statements accurately reflects the challenges in gene therapy for emphysema?

Targeting lung tissues for gene delivery remains a significant hurdle. Signup and view all the answers

Which of the following is NOT a method commonly used in gene therapy?

Blood transfusion as a means to deliver genetic material. Signup and view all the answers

Which is an incorrect assertion concerning gene therapy for Severe Combined Immunodeficiency (SCID)?

This therapy has been shown to be ineffective in young children. Signup and view all the answers

Which of the following statements is incorrect regarding gene therapy's potential for treating monogenic diseases?

It is guaranteed that gene therapy will provide a permanent cure for all patients. Signup and view all the answers

Which of the following is a limitation of current gene therapy approaches for monogenic diseases?

The risk of immune reactions against viral vectors. Signup and view all the answers

Which of the following approaches does NOT enhance the efficacy of gene therapy?

Regularly altering the genetic material being introduced. Signup and view all the answers

Which of the following statements about oncolytic agents is correct?

Oncolytic agents are genetically engineered viruses that specifically target cancer cells. Signup and view all the answers

Which of the following best describes the role of adenovirus in gene therapy?

It is the most commonly used viral vector for gene transfer in clinical trials. Signup and view all the answers

Which of the following concerning gene transfer is incorrect?

Gene transfer techniques solely rely on bacterial vectors. Signup and view all the answers

Which of the following is a potential ethical concern regarding gene therapy?

Access to gene therapy might not be equitable. Signup and view all the answers

Which statement about cancer cell targeting by oncolytic vectors is incorrect?

Herpes simplex virus type I is ineffective against cancer cells. Signup and view all the answers

Which of the following is NOT a method by which oncolytic agents induce cell death?

Enhancement of cancer cell metabolism Signup and view all the answers

Which of the following statements about the effects of using retroviral vectors is incorrect?

The long-term effects of retroviral vectors are well-studied and fully understood. Signup and view all the answers

Which of the following accurately describes suicide genes in gene therapy?

They cause cellular death upon expression. Signup and view all the answers

Which of the following is not a reason for choosing specific viruses in oncolytic therapy?

Cost-effectiveness in production. Signup and view all the answers

Which of the following concerns is least related to the introduction of gene therapy?

The ability to genetically enhance non-cancerous individuals. Signup and view all the answers

Study Notes

Biochemistry and Biotechnology Fundamentals: Gene Therapy II

This course covers gene therapy approaches for various genetic disorders.

Gene therapy for monogenic diseases
Gene therapy for cystic fibrosis
Gene therapy for emphysema
Gene therapy for sickle cell anemia
Gene therapy for SCID

Learning Objectives

Explain gene therapy approaches for cystic fibrosis
Describe alpha-1 antitrypsin deficiency and its treatment
Differentiate between different gene therapy approaches for treating sickle cell anemia
Discuss the reasons and therapy for SCID

Gene Therapy: Overview of ex vivo Gene Therapy

Cells are harvested from the patient
A virus is altered so it cannot reproduce
Altered cells are injected into the patient's body
Cells become transgenic
Altered cells produce the desired protein
Altered virus is mixed with patient's cells
A gene is inserted into the virus

Cystic Fibrosis (Monogenic Disease)

Genetic CFTR mutation at birth
CFTR mutant protein with abnormal ion transport
Viscous mucus and inflammation
Leads to infection, obstruction, inflammation and structural damage
Resulting in pulmonary insufficiency, premature death, and decreased mucus viscosity.
Treatments include rhDNase I for improved pulmonary function and reduced infections.

Cystic Fibrosis (Monogenic Disease): Mechanism

Healthy cells have a functional CFTR protein for Cl− ion transport.
In cystic fibrosis, CFTR protein is faulty, leading to thick mucus build-up.
Mucus builds up in the lungs, pancreas, and other organs.

Gene Therapy for Cystic Fibrosis

Correct version of the CFTR gene is placed in the patient's cells
The presence of the correct CFTR gene allows cells to produce normal CFTR proteins.
Gene therapy for cystic fibrosis can be achieved through:
- Non-integrating gene therapy
- Integrating gene therapy

Non-Integrating Gene Therapy for Cystic Fibrosis

A piece of DNA with the correct CFTR gene is provided to the patient's cells
The DNA remains separate from the patient's genome
Cells can still use the new copy to make normal CFTR proteins.
Advantage: doesn't disrupt the rest of the genome.
Disadvantage: not permanent (repeated treatment may be needed).

Integrating Gene Therapy for Cystic Fibrosis

A piece of DNA containing the correct CFTR gene is delivered to patient's cells
The new gene becomes a permanent part of the patient's genome.
Advantage: permanent.
Disadvantage: the new gene could insert into a critical part of the genome.

Disadvantages of Using Adenovirus in CF Gene Therapy

Lack of adenovirus receptor on alveolar sac cells leading to low transduction
Immunogenicity (immune response)
Non-integration: adenovirus genome stays as an episome and doesn't integrate into host genome.
Not permanent

Emphysema

Emphysema is a lung disease primarily caused by smoking, air pollution, chemical fumes, and exposure to irritants damaging the lungs and airways.
Symptoms include shortness of breath, coughing, and fatigue.
Alpha-1 antitrypsin deficiency (AATD) can also cause emphysema.

Alpha-1 Antitrypsin Deficiency

Elastin is a stretchy protein crucial for lung function.
Elastase, a protein that degrades elastin, needs control.
Alpha-1-antitrypsin (AAT) inhibits elastase to prevent elastin breakdown.
In AATD, low AAT levels lead to excessive elastase activity and lung damage, causing emphysema.

Gene Therapy for Alpha-1 Antitrypsin Deficiency

A short coding sequence for AAT would correct the deficiency.
Gene delivery could be targeting various tissues and cell types, without impacting lungs.
The goal of gene therapy is to continuously express the gene to treat affected individuals

Sickle Cell Anemia (Monogenic Disease)

In sickle cell anemia, the amino acid glutamic acid is replaced by valine in the beta-globin chain of hemoglobin
Sickle cells block blood flow, depriving organs of oxygen
This can damage nerves and organs, including kidneys, liver, and spleen.
Leads to symptoms like anemia, jaundice, pain crises, splenomegaly, and stroke.

Sickle Cell Anemia: Gene Therapy

Several gene therapy approaches exist for sickle cell anemia.
Using CRISPR, a gene is silenced to enable production of fetal hemoglobin, preventing sickling

Severe Combined Immunodeficiency (SCID)

A rare genetic disorder where development of functional T cells and B cells is impaired.
This is due to multiple genetic mutations, with two main types of lymphocytes being B and T cells
This results in an almost absent immune system.
Adenosine deaminase (ADA) deficiency is a common cause because the buildup of dATP inhibits ribonucleotide and DNA synthesis, thereby impacting T and B cells, which are vulnerable as they are rapidly dividing cells.
Gene therapy approaches are available

ADA Converts Deoxyadenosine to a Non-Toxic Substance

Normal function of ADA converts deoxyadenosine into deoxyinosine (not toxic)
ADA deficiency leads to deoxyadenosine buildup that's harmful, damaging B and T cells

Gene Therapy for SCID: Ex Vivo Approach

Hematopoietic stem cells (HSCs) are extracted.
Modified HSCs are introduced and returned to the bloodstream
Viral vectors like lentiviruses and retroviruses allow for long-term gene expression.

Cancer Gene Therapy: Immunotherapy

Cancer cells are harvested and grown in vitro.
Genes are added to make cancer cells more recognizable by the immune system.
Altering the cells is followed by growing and killing the cells in vitro
Cellular contents are used to create a vaccine

Cancer Gene Therapy: Oncolytic Agents

Gene therapy uses viruses that are modified to target and destroy cancer cells.
These modified viruses propagate themselves and/or express cytotoxic proteins, causing cancer cell death.

Cancer Gene Therapy: Gene Transfer

The introduction of foreign genes into cancer cells or surrounding tissue.
Genes can be suicide genes or antiangiogenesis genes.
Adenovirus is commonly used as a gene transfer viral vector in clinical trials.

Ethical considerations in gene therapy

Carcinogenesis, interference with natural selection, effect on offspring, access to therapy, use of gene therapy for enhancement, prenatal use, distinction between good and bad uses of technology, who decides on normal and disordered traits, high costs of gene therapy, and widespread use altering social acceptance of individuals.

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Description

Test your knowledge on gene therapy methods for Cystic Fibrosis and the characteristics of emphysema. This quiz covers key statements regarding adenoviruses, non-integrating methods, and symptoms associated with emphysema. Challenge yourself with these important medical concepts!

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