Gene Therapy I Quiz
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Questions and Answers

Which pair of nucleotide bases bond together in DNA?

  • G with T
  • A with C
  • C with A
  • A with T (correct)

    • Human DNA consists of approximately 2 billion base pairs.

    False

    What are the two types of sex chromosomes in humans?

    XX in females and XY in males

    The two strands of DNA are held together by _____ bonds.

    <p>hydrogen</p> Signup and view all the answers

    Match the terms with their definitions:

    <p>Chromosome = Long linear pieces of DNA Gene = Instructions for protein synthesis Nucleotide = Building blocks of DNA Base Pair = Two nucleotides bonded together</p> Signup and view all the answers

    How many pairs of chromosomes are typically found in human cells?

    <p>23</p> Signup and view all the answers

    All genes in the human genome code for proteins.

    <p>False</p> Signup and view all the answers

    What is the approximate number of protein-coding genes in the human reference genome?

    <p>20,000 to 25,000</p> Signup and view all the answers

    A chromosome consists of proteins called _____ and DNA.

    <p>histones</p> Signup and view all the answers

    Which of the following statements is true regarding autosomes?

    <p>There are 22 pairs of autosomes.</p> Signup and view all the answers

    What is the primary location of most DNA in a cell?

    <p>Nucleus</p> Signup and view all the answers

    Mitochondrial DNA is found exclusively in the cell nucleus.

    <p>False</p> Signup and view all the answers

    What is an organism's complete set of nuclear DNA called?

    <p>genome</p> Signup and view all the answers

    Each molecule of DNA is a double helix formed from two complementary strands known as __________.

    <p>strands</p> Signup and view all the answers

    Which of the following is NOT a method of gene delivery?

    <p>Transcription factor modulation</p> Signup and view all the answers

    Match the following gene delivery methods with their descriptions:

    <p>Viral vectors = Use of viruses to deliver genetic material Liposomes = Spheres made of lipid bilayers that encapsulate DNA Calcium phosphate transfection = A chemical method to introduce DNA into cells Electroporation = Use of an electric field to increase cell permeability for DNA entry</p> Signup and view all the answers

    Gene therapy can only use viral vectors for delivering genes.

    <p>False</p> Signup and view all the answers

    Describe one potential approach used in gene therapy.

    <p>Replacing a faulty gene with a functional one.</p> Signup and view all the answers

    What type of genetic disorder is caused by a mutation in one single gene?

    <p>Monogenic disorders</p> Signup and view all the answers

    Polygenic diseases are influenced by the combined effects of many genes.

    <p>True</p> Signup and view all the answers

    What is a mutation?

    <p>A change in the DNA sequence of a cell.</p> Signup and view all the answers

    A _____ mutation is a nucleotide substitution that does not change the amino acid sequence.

    <p>silent</p> Signup and view all the answers

    Match the types of mutations with their descriptions:

    <p>Silent mutation = Causes no change in amino acid Missense mutation = Results in a different amino acid Nonsense mutation = Creates a premature stop codon Frameshift mutation = Shifts the reading frame of the sequence</p> Signup and view all the answers

    Which of the following is NOT a type of mutation?

    <p>Dominant mutation</p> Signup and view all the answers

    Gene therapy can be used to cure genetic diseases by replacing defective genes.

    <p>True</p> Signup and view all the answers

    Name one factor that can cause mutations.

    <p>Environmental agents, such as chemicals or radiation.</p> Signup and view all the answers

    Heart disease and diabetes are examples of _____ diseases.

    <p>polygenic</p> Signup and view all the answers

    Which of the following best describes a missense mutation?

    <p>It results in a different amino acid being incorporated.</p> Signup and view all the answers

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

    <p>In vivo gene therapy delivers corrected genes directly to the patient.</p> Signup and view all the answers

    Ex vivo gene therapy involves modifying cells within the patient's body.

    <p>False</p> Signup and view all the answers

    What are vectors in the context of gene therapy?

    <p>Vectors are vehicles that carry genetic material and introduce it into target cells.</p> Signup and view all the answers

    In gene therapy, corrected genes can be given directly to the patient through __________ delivery or intravenous infusion.

    <p>local</p> Signup and view all the answers

    Match the type of gene therapy with its correct description:

    <p>In vivo = Directly administering corrected genes into the patient's body Ex vivo = Modifying cells outside the body before returning them to the patient Integrating vectors = Vectors that can integrate their genetic material into the host DNA Non-integrating vectors = Vectors that do not integrate their genetic material into the host DNA</p> Signup and view all the answers

    Which type of therapy is often used for blood disorders?

    <p>Ex vivo gene therapy</p> Signup and view all the answers

    What happens to the viral genome in viral vectors used for gene therapy?

    <p>The viral genome is replaced by the gene therapy transgene.</p> Signup and view all the answers

    What is the primary goal of gene therapy?

    <p>To repair or compensate for faulty genes</p> Signup and view all the answers

    Gene therapy is most effective for polygenic disorders.

    <p>False</p> Signup and view all the answers

    What role do vectors play in gene therapy?

    <p>Vectors transfer genetic material into target cells.</p> Signup and view all the answers

    The _____ are often used as vectors in gene therapy due to their ability to efficiently enter cells.

    <p>viruses</p> Signup and view all the answers

    Match the following mechanisms of gene therapy with their functions:

    <p>Reducing disease-causing proteins = Decreases harmful proteins Increasing disease-fighting proteins = Boosts defense mechanisms Producing modified proteins = Creates altered proteins for functionality</p> Signup and view all the answers

    Which of the following describes a potential outcome of successful gene therapy?

    <p>Reduction in disease symptoms</p> Signup and view all the answers

    In gene therapy, the original viral genes remain intact within the vector.

    <p>False</p> Signup and view all the answers

    What specific type of disorders is gene therapy likely to be most useful for?

    <p>Monogenic disorders.</p> Signup and view all the answers

    Gene therapy corrects faulty genes by producing new or _____ proteins.

    <p>modified</p> Signup and view all the answers

    What is a common method for delivering genes into cells in gene therapy?

    <p>Using a carrier known as a vector</p> Signup and view all the answers

    Study Notes

    Course Information

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

    Contents

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

    Learning Objectives

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

    Levels of Organization

    • Cell
    • Tissue
    • Organ
    • Organ system
    • Organism

    DNA Structure

    • Located in the cell nucleus (nuclear DNA) and mitochondria (mitochondrial DNA or mtDNA)
    • An organism's complete set of nuclear DNA is called its genome
    • A double helix formed from two complementary strands
    • Made up of four nucleotide bases: adenine (A), thymine (T), guanine (G), and cytosine (C)
    • Nucleotides attach to each other (A with T, and G with C) to form base pairs
    • The human genome consists of approximately 3 billion base pairs
    • Strands held together by hydrogen bonds

    Chromosome

    • Nuclear DNA is in the form of long, linear pieces called chromosomes
    • Found inside the nucleus of a cell
    • Composed of proteins (histones) and DNA
    • Each cell typically contains 23 pairs of chromosomes
    • 22 pairs are autosomes, and 1 pair are sex chromosomes (XX in females, XY in males)

    Human Genome

    • 23 pairs of chromosomes
    • 3 billion base pairs
    • 30,000 genes

    Genes

    • Made up of DNA
    • Act as instructions to make proteins
    • Some genes don't code for proteins; instead, they help control other genes
    • Passed from parents to offspring
    • Contain information for physical and biological traits
    • Human reference genome contains approximately 20,000-25,000 protein-coding genes
    • Only about 1-2% of the entire genome codes for proteins

    Types of Genetic Disorders

    • Monogenic disorders: caused by mutations in a single gene (e.g., cystic fibrosis)
    • Polygenic disorders: caused by the combined effects of multiple genes (e.g., heart disease, diabetes)

    Mutations

    • Any change in the DNA sequence of a cell
    • Can be caused by mistakes during cell division or environmental factors (e.g., viral infection, chemicals, radiation)
    • Can be harmful, beneficial, or have no effect
    • Types include silent, missense, nonsense, and frameshifts

    Gene Therapy

    • Technique using genes to treat, prevent or cure diseases.
    • Often involves adding new gene copies, replacing defective genes, or introducing healthy gene versions
    • Can be used to repair genes or compensate for losses
    • Likely most useful for treating monogenic disorders

    Gene Therapy Mechanism of Action

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

    Gene Therapy Drug Development

    • Starts with a specific therapeutic agent
    • Skips the drug discovery phase
    • 8-10 years for development
    • Cost: $0.6 billion

    Gene Therapy Workings (Methods)

    • Gene replacement: Replacing a faulty gene with a healthy one
    • Gene silencing: Inactivating a mutated gene that causes problems
    • Gene addition: Introducing a new gene to impact disease state
    • Gene editing: Permanently manipulating a gene in the genome

    Gene Delivery Mechanisms

    • Uses a "vector" (carrier) to transfer genetic material into the target cell
    • Viral vectors are commonly used because they efficiently enter cells. Viral genes are removed and replaced by target genes
    • Once inside the cell, the gene makes functional proteins or targets disease factors

    Delivery Strategies

    • In vivo: Corrected genes are introduced directly into the patient (e.g., through an IV or local delivery)
    • Ex vivo: Cells are removed, modified outside the body, then returned to the patient (e.g., hematopoietic stem cell therapies)

    Vectors (Gene Delivery Vehicles)

    • Viral vectors: Retroviruses, Lentiviruses, Adenoviruses, Adeno-associated viruses
    • Non-viral vectors: Liposomes
    • Types differ in integration ability (integrating or non-integrating)

    Integrating Vectors

    • Integrate into the host DNA.
    • Can potentially induce the activation of oncogenes or disrupt tumor suppressor genes, leading to cancer.
    • Primarily for ex vivo gene therapy

    Non-Integrating Vectors

    • Do not integrate into the host DNA
    • Provide short-term expression.
    • Lower probability of insertional mutagenesis and cancer but higher risk of triggering an immune response

    Viral Vectors - Characteristics

    • High transfection efficiency
    • Often used to introduce genes into target cells
    • Can integrate into the host genome
    • Possible immune reactions
    • Packaging limitations (carrying capacity for gene payload)

    Non-Viral Vectors - Characteristics

    • Lower transfection efficiency
    • Fewer immune reactions
    • Rarely integrate into the host genome
    • Larger packaging capacity than viral vectors
    • Often used in in vivo gene therapy settings

    Gene Therapy Approaches

    • Gene augmentation therapy: Adding a healthy gene to correct a faulty one
    • Gene inhibition therapy: Stopping the function of a faulty gene, often one that's overactive
    • Killing specific cells: Destroying cells with mutated genes (e.g., cancer cells)

    Challenges for Gene Therapy Development

    • Safety/Immunity: Immune response to transgene or vector, Immune suppression
    • Manufacturing: Cost, Demand, Long-term effects, Uncertainty how long therapeutic benefit lasts, Limited population for rare disease trials

    Benefits of Gene Therapy

    • Precise medicine targeting the cause of disease
    • Potential for single administration
    • Can reduce or eliminate the need for other treatments
    • Treatment of neurological conditions

    Requirements of Ideal Gene Therapy

    • Targeting specific cells
    • Lasting effects
    • Controlling immune responses
    • Not disrupting other normal genes

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

    Description

    Test your knowledge on the fundamentals of gene therapy, including mechanisms of action, delivery strategies, and the various vehicles used in gene therapy. Dive into the molecular details of DNA structure and gene delivery vectors that are pivotal in biotechnology. Perfect for students of Biochemistry and Biotechnology.

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