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 (B)

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

All genes in the human genome code for proteins.

<p>False (B)</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. (D)</p> Signup and view all the answers

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

<p>Nucleus (D)</p> Signup and view all the answers

Mitochondrial DNA is found exclusively in the cell nucleus.

<p>False (B)</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 (C)</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 (B)</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 (D)</p> Signup and view all the answers

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

<p>True (A)</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 (B)</p> Signup and view all the answers

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

<p>True (A)</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. (D)</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. (C)</p> Signup and view all the answers

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

<p>False (B)</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 (C)</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 (C)</p> Signup and view all the answers

Gene therapy is most effective for polygenic disorders.

<p>False (B)</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 (C)</p> Signup and view all the answers

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

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

Flashcards

DNA location

Most DNA is located in the cell nucleus, where it is called nuclear DNA. However, a small amount can also be found in the mitochondria, where it is called mitochondrial DNA or mtDNA.

Genome

The complete set of nuclear DNA in an organism is called its genome.

DNA structure

Each DNA molecule is a double helix formed from two complementary strands.

In Vivo Gene Therapy

A type of gene therapy where corrected genes are directly delivered to the patient's cells within their body, often through an IV or local injection.

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Ex Vivo Gene Therapy

Cells are removed from the patient, genetically modified in a lab (e.g., adding a new gene or correcting a faulty one), and then returned to the patient.

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Viral Vectors

Naturally occurring viruses that are genetically modified to carry therapeutic genes and deliver them into target cells.

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Transgene

The therapeutic gene inserted into a viral vector for delivery and expression in target cells.

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Integrating Viral Vectors

Viral vectors that permanently insert their genetic material into the host cell's DNA.

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Non-Integrating Viral Vectors

Viral vectors that do not integrate their genetic material into the host cell's DNA; the transgene remains separate.

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Hematopoietic Stem Cell Therapies

Ex vivo gene therapy approaches using blood stem cells, often used to treat blood disorders.

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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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How do DNA strands connect?

The nucleotides attach to each other in pairs: adenine with thymine (A-T) and guanine with cytosine (G-C). These pairs form chemical bonds called base pairs.

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What is a base pair?

A base pair is a pair of nucleotides in DNA that are linked together by hydrogen bonds. Adenine pairs with thymine, and cytosine pairs with guanine.

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How many base pairs are in the human genome?

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

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What are chromosomes?

Chromosomes are long, linear pieces of DNA found in the nucleus of a cell.

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What is a gene?

A gene is a segment of DNA that contains the instructions for making a specific protein, which is essential for bodily functions.

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What is the role of non-coding genes?

Some genes don't code for proteins; instead, they help regulate other genes.

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What is unique about chromosomes in humans?

Each human cell normally contains 23 pairs of chromosomes: 22 pairs are called autosomes, and one pair is called sex chromosomes (XX for females, XY for males).

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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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What percentage of the genome codes for proteins?

Only about 1-2% of the entire human genome (~3 billion base pairs) actually codes for proteins.

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Monogenic Disorder

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

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Polygenic Disorder

A disease influenced by the combined effects of multiple genes.

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Mutation

Any change in the DNA sequence of a cell.

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Causes of Mutations

Mutations can be caused by errors during cell division or exposure to DNA-damaging agents like viruses, chemicals, or radiation.

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Silent Mutation

A nucleotide substitution that doesn't change the amino acid coded for, so no change in protein function.

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Gene Therapy

A technique using genes to treat, prevent, or cure diseases by replacing defective or missing genes with healthy versions.

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Gene Therapy: Replacing a Defective Gene

Gene therapy can replace a faulty gene with a working copy, restoring normal function.

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Gene Therapy: Adding New Copies of a Gene

Gene therapy can introduce new copies of a gene to compensate for a missing or non-functional gene.

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Gene Therapy Goal

Gene therapy aims to correct underlying genetic defects that cause disease.

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Gene Therapy: Potential for Curing Diseases

Gene therapy holds promise for curing inherited diseases and other conditions that are currently difficult to treat.

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What is gene therapy?

Gene therapy aims to repair or replace faulty genes in cells to treat diseases.

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How does gene therapy work?

Gene therapy delivers healthy genetic material into cells, which either corrects faulty genes or provides missing genes.

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What are monogenic disorders?

Monogenic disorders are diseases caused by a single gene mutation.

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Why is gene therapy promising for monogenic disorders?

Gene therapy has the potential to directly target the faulty gene responsible for these disorders.

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What are vectors in gene therapy?

Vectors are carriers that deliver genetic material into cells.

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Why are viruses used as vectors in gene therapy?

Viruses have evolved to efficiently enter cells, making them good carriers for gene delivery.

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How are viruses modified for gene therapy?

Viruses are modified by removing their disease-causing genes and replacing them with therapeutic genes.

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How does gene therapy affect protein production?

Gene therapy can either reduce harmful protein production or increase production of beneficial proteins.

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What are the potential benefits of gene therapy?

Gene therapy could potentially cure or treat various diseases by repairing or replacing faulty genes.

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What are the potential risks of gene therapy?

Potential risks include immune reactions, uncontrolled cell growth, and off-target effects.

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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 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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