L4. Gene Therapy I - AI MCQs
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Questions and Answers

Which statement about in vivo gene therapy is correct?

  • In vivo gene therapy delivers corrected genes directly into the bloodstream.
  • In vivo gene therapy uses a viral vector to introduce new genes into patient cells. (correct)
  • In vivo gene therapy can only be administered via oral routes.
  • In vivo gene therapy involves altering genes in cultured cells.

Which description of ex vivo gene therapy is incorrect?

  • Ex vivo gene therapy is always performed using viral vectors. (correct)
  • This therapy typically involves hematopoietic stem cell modification.
  • In this approach, the patient's own cells are modified and then reintroduced.
  • Ex vivo gene therapy alters cells outside the body before returning them.

Which of the following accurately describes viral vectors used in gene therapy?

  • Viral vectors have no capacity to integrate with host DNA.
  • All viral vectors used are naturally occurring without modifications.
  • Viral vectors can only be non-integrating for gene therapy.
  • The viral genome is replaced with therapeutic genes for gene transfer. (correct)

Which statement about integrating and non-integrating viral vectors is incorrect?

<p>Integrating vectors typically lead to transient expression of the gene. (C)</p> Signup and view all the answers

Which of the following statements regarding gene delivery vehicles is incorrect?

<p>Only bacterial vectors can be used for gene delivery in gene therapy. (C)</p> Signup and view all the answers

Which of the following accurately describes monogenic diseases?

<p>A single gene mutation can lead to a genetic disease. (D)</p> Signup and view all the answers

Which statement about polygenic diseases is correct?

<p>They involve the interaction of multiple genetic factors. (A)</p> Signup and view all the answers

Which of the following statements about mutations is incorrect?

<p>Mutations can only be harmful to an organism. (D)</p> Signup and view all the answers

Which of the following types of mutations does not affect protein function?

<p>Silent mutations (A)</p> Signup and view all the answers

Which of the following statements regarding gene therapy is correct?

<p>It can treat or prevent diseases using healthy gene copies. (B)</p> Signup and view all the answers

Which characteristic is not true about the nature of mutations?

<p>All mutations result in an observable change in phenotype. (D)</p> Signup and view all the answers

Which of the following best describes a missense mutation?

<p>It changes one amino acid in a protein sequence. (A)</p> Signup and view all the answers

Which of the following statements about DNA damage and mutations is incorrect?

<p>DNA damage is always repaired properly by the cell. (C)</p> Signup and view all the answers

Which statement about the base pairing in DNA is correct?

<p>Cytosine pairs with guanine. (B)</p> Signup and view all the answers

Which of the following pairs describes incorrect information about chromosomes?

<p>All chromosomes are linear and reside in the cytoplasm. (B)</p> Signup and view all the answers

Which statement regarding genes is incorrect?

<p>All genes code for proteins directly. (A)</p> Signup and view all the answers

Which of the following statements about the human genome is correct?

<p>1-2% of the human genome actually codes for proteins. (B)</p> Signup and view all the answers

Which statement is incorrect regarding nucleotides?

<p>Base pairs are formed through covalent bonds between nucleotides. (A)</p> Signup and view all the answers

Which of the following statements about autosomes is correct?

<p>There are 22 pairs of autosomes in human cells. (C)</p> Signup and view all the answers

Which statement about the structure of DNA is correct?

<p>Hydrogen bonds between base pairs hold two DNA strands together. (A)</p> Signup and view all the answers

Which of the following statements regarding the functions of genes is incorrect?

<p>All genes have a direct impact on physical traits. (D)</p> Signup and view all the answers

Which statement about the human reference genome is incorrect?

<p>Protein-coding genes make up 10% of the genome. (B)</p> Signup and view all the answers

Which statement about the location of DNA in eukaryotic organisms is correct?

<p>Nuclear DNA is complemented by mitochondrial DNA. (C)</p> Signup and view all the answers

Which description of gene therapy vehicles is incorrect?

<p>Plasmids are unviable carriers for gene delivery. (D)</p> Signup and view all the answers

Which mechanism utilized in gene therapy is scientifically accurate?

<p>Gene addition seeks to replace mutated genes with healthy ones. (D)</p> Signup and view all the answers

Which strategy for gene delivery is incorrect?

<p>Viral transduction guarantees permanent gene expression in all cells. (B)</p> Signup and view all the answers

Which characteristic of nuclear DNA is false?

<p>It can vary significantly between individual cells in a multicellular organism. (A)</p> Signup and view all the answers

Which of the following statements about gene therapy approaches is incorrect?

<p>Gene therapy exclusively uses viral vectors for effective delivery. (C)</p> Signup and view all the answers

Which statement is correct regarding the use of adeno-associated virus (AAV) in gene therapy?

<p>AAV serves as a vector to deliver a healthy version of a mutated gene. (A)</p> Signup and view all the answers

Which statement about mitochondrial DNA (mtDNA) is accurate?

<p>mtDNA contributes to the overall genome of an organism. (C)</p> Signup and view all the answers

Which implication of gene therapy is misleading?

<p>Gene therapy permanently eliminates all health issues. (B)</p> Signup and view all the answers

Which of the following statements about gene inhibition therapy is accurate?

<p>It introduces a gene product that interferes with either the overactive gene or its product. (D)</p> Signup and view all the answers

Which assertion about the genome is false?

<p>Genome size is consistent across all species. (D)</p> Signup and view all the answers

What is the main goal of killing specific cells in gene therapy?

<p>To selectively destroy certain diseased cells, like cancer cells. (B)</p> Signup and view all the answers

Which of the following best describes the method of cell death induced by gene therapy?

<p>Cell death is controlled and can involve inducing apoptosis. (D)</p> Signup and view all the answers

Which is an essential aspect of inserting DNA into disease-affected cells?

<p>The DNA must be specific to the diseased cells being treated. (A)</p> Signup and view all the answers

In which scenario is gene inhibition therapy particularly useful?

<p>When genes become overactive and promote uncontrolled cell division. (B)</p> Signup and view all the answers

Which of the following describes a method used in killing specific cells during gene therapy?

<p>Inserting DNA that produces a toxic product leading to apoptosis. (B)</p> Signup and view all the answers

What potential risk is associated with gene therapy that involves killing specific cells?

<p>It can inadvertently kill healthy cells if not targeted correctly. (B)</p> Signup and view all the answers

What is the primary characteristic of the adeno-associated virus (AAV) used as a vector?

<p>It is nonpathogenic and does not cause disease. (D)</p> Signup and view all the answers

Which of the following statements about in vivo gene therapy is incorrect?

<p>In vivo gene therapy occurs entirely outside the patient's body. (A)</p> Signup and view all the answers

Which statement regarding ex vivo gene therapy is correct?

<p>Cells modified in ex vivo gene therapy are replaced in the patient. (A)</p> Signup and view all the answers

Which of the following is characteristic of integrating viral vectors?

<p>Integrating viral vectors permanently alter the host's genetic material. (A)</p> Signup and view all the answers

Which statement regarding the use of viral vectors in gene therapy is incorrect?

<p>Genetically modified viruses used in gene therapy are typically capable of causing diseases. (C)</p> Signup and view all the answers

Which of the following statements concerning vectors used for gene delivery is incorrect?

<p>Non-integrating vectors integrate genetic material into the host's DNA. (B)</p> Signup and view all the answers

Which statement about monogenic disorders is correct?

<p>They result from mutations in a single gene. (C)</p> Signup and view all the answers

Which assertion regarding mutations is incorrect?

<p>Mutations can only be harmful to the organism. (B)</p> Signup and view all the answers

Which statement about the effects of mutations is incorrect?

<p>All mutations lead to changes in amino acid sequences. (A)</p> Signup and view all the answers

Which statement about gene therapy techniques is incorrect?

<p>Gene therapy can only add new genes to the patient's cells. (C)</p> Signup and view all the answers

Which of the following statements about the nature of mutations is incorrect?

<p>All mutations are detectable through standard testing methods. (A)</p> Signup and view all the answers

Which statement regarding the mechanisms of gene therapy is misleading?

<p>Gene therapy can exclusively use synthetic genes. (C)</p> Signup and view all the answers

Which statement about the role of environmental factors in mutations is incorrect?

<p>All mutations are inherited and not influenced by environment. (D)</p> Signup and view all the answers

Which of the following accurately describes the purpose of gene inhibition therapy?

<p>It stops the function of a gene that is behaving improperly. (D)</p> Signup and view all the answers

Which statement about the use of adenoviruses in gene therapy is incorrect?

<p>They can cause disease in humans. (B)</p> Signup and view all the answers

Which of the following statements about apoptosis in gene therapy is correct?

<p>Apoptosis is a process that can be initiated artificially. (B)</p> Signup and view all the answers

Which of the following describes a characteristic of the gene therapy technique that aims to kill specific cells?

<p>It requires the inserted DNA to be targeted carefully. (B)</p> Signup and view all the answers

Which statement about the adeno-associated virus (AAV) is correct?

<p>AAV can be used as a vector for gene delivery. (D)</p> Signup and view all the answers

Which of the following correctly represents an application of gene therapy aimed at cancer treatment?

<p>Implanting DNA that produces immune responses against cancer cells. (B)</p> Signup and view all the answers

Which statement regarding the gene therapy approach that involves killing specific cells is incorrect?

<p>It is often irrelevant in treating cancer. (A)</p> Signup and view all the answers

Which of the following is an incorrect implication of using gene therapy to modify cell behavior?

<p>All gene therapies are guaranteed safe with no side effects. (A)</p> Signup and view all the answers

Which statement about the production of toxic products in gene therapy is correct?

<p>Toxic products are utilized to provoke apoptosis in specific cells. (C)</p> Signup and view all the answers

Which of the following statements about the location of DNA in eukaryotic organisms is correct?

<p>Mitochondrial DNA is found in mitochondria and is distinct from nuclear DNA. (A)</p> Signup and view all the answers

Which of the following descriptions about gene delivery vectors is incorrect?

<p>Non-viral vectors are inherently less efficient than viral vectors in all scenarios. (A)</p> Signup and view all the answers

Which statement about gene therapy approaches is incorrect?

<p>Gene therapy is solely focused on correcting mutations rather than delivering genes. (D)</p> Signup and view all the answers

Which statement about viral vectors used in gene therapy is misleading?

<p>All viral vectors integrate their genetic material into the host genome. (D)</p> Signup and view all the answers

Which of the following claims about gene therapy mechanisms is incorrect?

<p>Only traditional methods can achieve targeted gene delivery. (D)</p> Signup and view all the answers

Which statement about the composition of chromosomes is incorrect?

<p>Chromosomes consist only of DNA without any proteins. (D)</p> Signup and view all the answers

Which of the following statements about nucleotide pairing in DNA is correct?

<p>Adenine pairs with thymine and cytosine pairs with guanine. (C)</p> Signup and view all the answers

Which of the following statements regarding the mechanisms for gene delivery is incorrect?

<p>All gene delivery methods have the same efficiency across different tissues. (D)</p> Signup and view all the answers

Which of the following is an incorrect description of genes?

<p>All genes code directly for the production of proteins. (C)</p> Signup and view all the answers

Which statement about the gene therapy mechanism of action is inaccurate?

<p>Gene therapy solely relies on repairing mutated DNA sequences. (C)</p> Signup and view all the answers

Which statement about the potential risks associated with gene therapy is correct?

<p>Immune responses can arise from gene therapy due to vector recognition. (A)</p> Signup and view all the answers

Which statement regarding the human reference genome is incorrect?

<p>The human genome consists of only 1 billion base pairs. (D)</p> Signup and view all the answers

Which of the following statements regarding gene delivery vehicles is misleading?

<p>Only retroviral vectors can incorporate genes into the host genome. (A)</p> Signup and view all the answers

Which of the following describes an incorrect function of genes?

<p>Some genes have no function and are completely redundant. (A)</p> Signup and view all the answers

Which statement about the makeup of DNA is incorrect?

<p>Thymine pairs with cytosine in the DNA structure. (B)</p> Signup and view all the answers

Which statement regarding the nature of genes is incorrect?

<p>The entirety of genes predominantly codes for protein structure. (B)</p> Signup and view all the answers

Which description of nucleotide bases is incorrect?

<p>Guanine forms a base pair with adenine. (D)</p> Signup and view all the answers

Flashcards

Monogenic disorder

A genetic disease caused by a mutation in a single gene.

Polygenic disorder

A genetic disease influenced by multiple genes.

Mutation

A change in DNA sequence of a cell.

Silent mutation

A nucleotide substitution that doesn't change the amino acid sequence or protein function

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Missense mutation

A mutation that changes one amino acid to another.

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Nonsense mutation

A mutation that changes a codon that specifies an amino acid to a stop codon.

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Frameshift mutation

A mutation that shifts the reading frame of the genetic code.

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Gene therapy

Using genes to treat, prevent, or cure a disease.

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In vivo gene therapy

Corrected genes are delivered directly to the patient's cells inside the body, often through an IV or local delivery to a specific organ, using a viral vector.

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Ex vivo gene therapy

Cells are removed from the patient, modified outside the body in a lab, and returned to the patient. Often used for hematopoietic stem cell therapies.

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Gene therapy vector

A vehicle that carries genetic material into target cells for gene transfer. Often, viruses are genetically modified for this purpose.

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Viral vector integration

Some viral vectors can integrate their genetic material into the host cell's DNA.

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Integrating vs. non-integrating vectors

Viral vectors are categorized by their ability to integrate into the host DNA, either permanently or not.

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Gene Therapy Mechanism

A method using genetic material to treat or prevent disease by introducing or modifying genes.

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Gene Delivery Mechanisms

Methods for moving genetic material into target cells.

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Gene Delivery Strategies

Planned methods for delivering genetic material, considering factors like the target cell and disease.

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Gene Delivery Vectors

Carrier molecules used to transport genetic material into cells

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Gene Therapy Approaches

Various strategies used in gene therapy, including gene addition and gene editing.

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Nuclear DNA

DNA located in the cell nucleus.

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Mitochondrial DNA (mtDNA)

Small amount of DNA found in mitochondria within the cell.

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Genome

An organism's complete set of nuclear DNA.

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DNA double helix

Two complementary DNA strands that form a twisted ladder-like structure.

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Complementary DNA strands

Two DNA strands that pair together through base-pairing rules.

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DNA Building Blocks

Four nucleotide bases (adenine, thymine, guanine, and cytosine) that form the structure of DNA.

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Base Pairs

Specific pairings of nucleotide bases (A with T, and G with C) that connect DNA strands.

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Human Genome Size

Approximately 3 billion base pairs of DNA.

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Chromosomes

Long, linear pieces of DNA found inside the nucleus of a cell, comprising proteins (histones) and DNA.

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Autosomes

Any chromosome that is not a sex chromosome. Humans have 22 pairs of autosomes

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Sex Chromosomes

The X and Y chromosomes that determine sex in humans (XX females and XY males).

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Protein-Coding Genes

Genes that contain instructions for making proteins.

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Human Protein-Coding Genes

Estimated to be between 20,000 and 25,000 protein-coding genes in the human reference genome.

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Genome Coding Proportion

Only about 1-2% of the human genome codes for proteins.

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Gene therapy using AAV vector

Healthy gene is packaged into a modified, harmless virus (AAV) for delivery to target cells. The virus acts as a carrier to correct the mutated gene.

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Gene inhibition therapy

This therapy stops the activity of a faulty gene by introducing a new gene that either blocks the faulty gene's function or its product.

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Gene inhibition therapy application

Useful for overactive genes, such as those causing uncontrolled cell division (like in cancer)

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Killing specific cells

This gene therapy technique targets and destroys cells with mutated genes, such as cancer cells.

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Cell death triggering method

Inserting DNA into cells causing them to produce a lethal substance or signaling to the immune system for destruction.

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Apoptosis

A programmed cell death, triggered by a specific cellular process.

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Immunotherapy in gene therapy

Using gene therapy to direct the immune system to attack diseased cells.

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Specificity in gene therapy

Crucial to avoid harming healthy cells when targeting diseased cells.

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Cancer cell targeting

Gene therapies frequently used to target and control the growth of cancer cells.

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What are viral vectors?

Viruses are genetically modified to carry genetic material and introduce it into target cells. Replacing the viral genome with the gene therapy transgene.

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What is the purpose of gene therapy?

Gene therapy aims to treat, prevent, or cure diseases by introducing or modifying genes.

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What are the building blocks of DNA?

DNA is made up of four nucleotide bases: adenine (A), thymine (T), guanine (G), and cytosine (C).

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What are base pairs?

Base pairs are chemical bonds that connect the two DNA strands. Adenine (A) pairs with thymine (T), and guanine (G) pairs with cytosine (C).

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How many base pairs in the human genome?

One copy of the human genome consists of approximately 3 billion base pairs of DNA.

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What are chromosomes?

Chromosomes are long, linear pieces of DNA found inside the nucleus of a cell. They are made up of proteins (histones) and DNA.

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How many chromosomes do humans have?

Each cell normally contains 23 pairs of chromosomes, totaling 46 chromosomes.

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What are autosomes?

Autosomes are any chromosome that is not a sex chromosome. Humans have 22 pairs of autosomes.

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What are sex chromosomes?

Sex chromosomes are the X and Y chromosomes that determine an individual's sex. Females have two X chromosomes (XX), while males have one X and one Y chromosome (XY).

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What are genes made of?

Genes are made up of DNA.

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What do genes do?

Some genes provide instructions for making proteins, which are essential for the body to function. Others help control other genes.

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How many protein-coding genes are in the human genome?

The human reference genome contains somewhere between 20,000 and 25,000 protein-coding genes.

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Gene Therapy Approach 1: Gene Correction

Replacing a mutated gene with a healthy copy to restore proper function. This is done by packaging a healthy gene into a harmless virus (AAV) that acts as a delivery vehicle.

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Gene Therapy Approach 2: Gene Inhibition

Stopping a faulty gene's activity by introducing a new gene that interferes with its function or its product. This helps to control overactive genes, such as those contributing to cancer.

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Gene Therapy Approach 3: Killing Diseased Cells

Targeting and destroying cells with mutated genes. This is often used in cancer treatment. Gene therapy can cause a cell to die by producing a toxic substance, triggering apoptosis (programmed cell death), or by signaling the immune system to attack.

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AAV (Adeno-Associated Virus)

A harmless virus that is engineered to act as a vector for gene delivery. It carries a healthy copy of a gene into cells.

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Overactive Genes

Genes that produce too much of a certain protein or function too much. This can contribute to diseases like cancer.

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Gene Therapy for Cancer

Gene therapy techniques can be used to kill cancer cells, stop the growth of tumors, or boost the immune system's ability to fight cancer.

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Study Notes

Course Information

  • Course Title: Biochemistry and Biotechnology Fundamentals, Gene Therapy I
  • Course Code: 1120-111
  • Instructor: Dr. Ahmed Hemdan
  • Department: Pharmaceutical Chemistry

Contents

  • Definitions
  • Gene therapy mechanism of action
  • Delivery mechanisms and strategies
  • Gene delivery vectors
  • Gene therapy approaches

Learning Objectives

  • Explain the mechanism of gene therapy
  • Describe the mechanisms for gene delivery and the strategies used
  • Differentiate between different gene therapy delivery vehicles
  • Discuss different approaches used in gene therapy

Levels of Organization

  • A diagram showing the hierarchical levels of organization – cell, tissue, organ, organ system, organism

DNA Structure

  • DNA is located in the nucleus (nuclear DNA) and mitochondria (mitochondrial DNA)
  • A complete set of nuclear DNA is called a genome
  • Each DNA molecule is a double helix made of two strands
  • Four nucleotide bases (adenine, thymine, guanine, cytosine) form the building blocks
  • The base pairs (A with T and G with C) connect two strands via hydrogen bonds

Chromosomes

  • Chromosomes are composed of DNA and proteins (histones)
  • A human cell typically has 23 pairs of chromosomes (44 autosomes and 2 sex chromosomes)

Genes

  • Genes are segments of DNA that carry instructions for protein synthesis
  • Many genes do not code for proteins, but regulate other genes.
  • The human genome contains between 20,000 and 25,000 protein-coding genes
  • Less than 2% of the genome codes for proteins (~3 billion base pairs total)

Types of Genetic Diseases

  • Monogenic diseases: caused by mutations in a single gene (e.g., cystic fibrosis)
  • Polygenic diseases: influenced by multiple genes (e.g., heart disease, diabetes)

DNA, Genes, and Chromosomes Summary

  • DNA is the fundamental genetic material, organized into chromosomes.
  • Genes, segments of DNA, hold genetic information for proteins.
  • Genetic diseases can be monogenic (single gene) or polygenic (multiple genes).

Mutations

  • A mutation is any change in the DNA sequence.
  • Mutations can be silent, missense, nonsense, or frameshift mutations.
  • Mutations can be harmful, beneficial, or have no effect.
  • Mutations can be caused by cell division errors or environmental factors (viruses, chemicals, radiation).

Gene Therapy

  • Gene therapy is a technique using genes to treat, prevent, or cure diseases.
  • Often involves adding new copies of a gene, replacing a defective gene, or modifying genes.
  • Somatic cell gene therapy affects the patient's cells but not future generations.
  • Germ-line gene therapy affects the patient's cells and future generations by correcting genes in germ line cells.

Gene Therapy Mechanism of Action

  • Once inside a cell, the agent corrects the faulty gene by:
    • Reducing disease-causing proteins
    • Increasing production of disease-fighting proteins
    • Producing new or modified proteins

Gene Therapy Delivery Strategies

  • In vivo: corrected genes are given directly to the patient (IV, local delivery to organs)
  • Ex vivo: cells are removed, modified, and returned to the patient. Examples include hematopoietic stem cell therapies for blood disorders.

Gene Delivery Vehicles (Vectors)

  • Viral vectors: naturally occurring viruses modified to carry therapeutic genes.
    • Examples include classes: Lentiviruses, Retroviruses, Adenoviruses, Adeno-associated viruses.
  • Nonviral vectors: synthetic molecules that deliver genetic material.
    • Examples include Liposomes
    • Newer technologies such as COVID-19 vaccines are using these techniques.

Integrating Viral Vectors

  • Integrate their genetic material into the host cell's DNA.
  • High probability of insertional mutagenesis, possibly causing cancer.
  • Often used for ex vivo therapies.

Non-Integrating Viral Vectors

  • Stable episomes that don't integrate into the genome.
  • Lower risk of causing insertional mutagenesis.
  • Commonly used for in vivo therapies.

Vector Types

  • Viral and non-viral vector types with their origins, transfection efficiency, immune reactions, and integration into DNA
  • Examples of some viral vectors: adenoviruses, adeno-associated viruses, retroviruses, and lentiviruses

Gene Therapy Approaches

  • Gene Augmentation: Corrects the effect of mutated genes via introducing a healthy copy of the gene (usually using adeno-associated viruses).
  • Gene Inhibition: Stops the function of faulty genes, either through interfering with the function of a faulty gene or through using their products.
  • Killing of Specific Cells: targeting diseased cells to be destroyed, causing cell death via apoptosis, or alerting the immune system.

Challenges for Gene Therapy Development

  • Safety/Immunology: immune response to the vector or transgene, immune suppression
  • Manufacturing: cost, demand
  • Long-term effects: uncertain benefit duration, potential side effects
  • Population size for rare diseases: limited population for testing and trials

Benefits of Gene Therapy

  • Precise targeting of the cause of a disease.
  • Potential for single administration of treatment.
  • Can eliminate the need for other treatments
  • Treatment of neurological conditions.

Requirements of Ideal Gene Therapy

  • Targeting specific cells
  • More lasting effects
  • Controlling immunity
  • Not disrupting other normal genes

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Gene Therapy I PDF - 1120-111 Biochemistry and Biotechnology Fundamentals

Description

Test your understanding of the fundamental principles of gene therapy, including its mechanisms, delivery strategies, and various approaches. This quiz covers essential topics such as gene delivery vectors and the hierarchical organization of biological systems.

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