Gene Therapy: Vectors and Ethical Concerns
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Questions and Answers

Which of the following best describes the primary goal of gene therapy?

  • To eliminate viruses from the body.
  • To replace faulty genes with functional copies to treat diseases. (correct)
  • To develop new types of vectors for gene delivery.
  • To enhance physical attributes like strength and speed.

Why are viruses often used as vectors in gene therapy?

  • Viruses are easily mass-produced and inexpensive.
  • Viruses can replicate the therapeutic gene within the patient's body.
  • Viruses are harmless to humans once their genetic material is removed.
  • Viruses have the natural ability to target specific cells and deliver genetic material. (correct)

In gene therapy aiming to correct an inherited form of blindness, what is the most likely target for the vector containing the corrected gene?

  • Brain cells.
  • Retinal cells. (correct)
  • Bone marrow cells.
  • Liver cells.

Which characteristic of retroviruses makes them suitable vectors for gene therapy?

<p>Their capacity to integrate their genetic material into the host cell's genome. (A)</p> Signup and view all the answers

What is a key difference between using retroviruses and adenoviruses as vectors in gene therapy?

<p>Retroviruses integrate their DNA into the host cell’s genome, while adenoviruses do not. (D)</p> Signup and view all the answers

A patient with cystic fibrosis is undergoing gene therapy. What would be the expected outcome if the therapy is successful?

<p>The patient's cells will produce functional proteins that mitigate the effects of cystic fibrosis. (A)</p> Signup and view all the answers

What is a significant challenge in gene therapy that researchers are actively working to overcome?

<p>Preventing the immune system from rejecting the introduced genes or vectors. (A)</p> Signup and view all the answers

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

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

What is a primary challenge in gene therapy when aiming to correct retinal defects?

<p>Guaranteeing the newly introduced DNA integrates permanently and safely into the patient's cells. (D)</p> Signup and view all the answers

Why is the precise placement of new genetic material important in gene therapy?

<p>To prevent the disruption of other essential bodily processes. (D)</p> Signup and view all the answers

What ethical concern arises from the potential of gene therapy to enhance human capabilities?

<p>The potential for abuse in creating 'designer babies' with selected traits. (B)</p> Signup and view all the answers

How does sperm sorting enable parents to select the gender of their child?

<p>By using a centrifuge to separate sperm carrying X and Y chromosomes based on weight. (A)</p> Signup and view all the answers

What distinguishes reproductive cloning from therapeutic cloning?

<p>Reproductive cloning intends to create a whole organism, while therapeutic cloning focuses on creating specific tissues or organs for healing. (C)</p> Signup and view all the answers

What ethical considerations are raised by therapeutic cloning?

<p>Whether the benefits of healing outweigh the potential for misuse or 'toying with nature'. (C)</p> Signup and view all the answers

What is a crucial step to ensure successful gene therapy, after getting the DNA into the right cells?

<p>Ensuring the DNA becomes a permanent part of the cell's genetic material and future cell divisions. (C)</p> Signup and view all the answers

What is a potential risk of fixing new DNA into a person's existing DNA during the process of gene therapy?

<p>Disrupting other essential processes within the body. (C)</p> Signup and view all the answers

Which of the following describes preimplantation genetic diagnosis?

<p>Testing embryos for genetic defects before implantation. (A)</p> Signup and view all the answers

What is an example of a future application of reproductive technology?

<p>Cloning organs to replace diseased ones. (B)</p> Signup and view all the answers

Flashcards

Gene Therapy

A treatment that replaces faulty genes with functional copies to treat diseases.

Vectors in Gene Therapy

Viral or non-viral agents used to deliver healthy genes to affected cells.

Types of Vectors

Includes retroviruses, adenoviruses, and herpes simplex viruses used in gene therapy.

Inherited Diseases

Disorders caused by defects in genes passed from parents to children.

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Sickle Cell Disease

A genetic disorder affecting hemoglobin in red blood cells, causing them to be misshapen.

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Cystic Fibrosis

A genetic condition affecting the lungs and digestive system, often due to a defective gene.

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Adenovirus

A type of virus used as a vector in gene therapy for delivering genetic material.

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Normal Retina DNA

Genetic material that could potentially restore vision by developing a retina.

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DNA Integration

The process of incorporating new DNA into a cell's genome.

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Permanent DNA Fix

Ensuring new DNA remains in the patient's cells for life.

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Safe DNA Insertion

Placing DNA in a way that does not disrupt other genes.

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Designer Babies

Babies genetically modified for desired traits.

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Sperm Sorting

Technique to select sperm for desired baby gender.

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Preimplantation Genetic Diagnosis

Testing embryos for genetic defects before implantation.

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Reproductive Cloning

Making a genetic copy of an entire organism.

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Therapeutic Cloning

Cloning specific tissues or organs for medical use.

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Ethical Considerations

Moral questions regarding the use of genetic technologies.

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Study Notes

Genetic Future: Potential and Ethical Concerns

  • Significant advancements in understanding DNA hold promise for altering disease treatment and human reproduction.
  • Gene therapy aims to replace faulty genes with functional ones, potentially eradicating inherited diseases.
  • Vectors, often viruses, deliver healthy genes to affected cells. Retroviruses, adenoviruses, adeno-associated viruses, herpes simplex viruses, liposomes, and naked DNA are types of vectors.
  • Challenges in gene therapy include delivering DNA to target cells, ensuring complete integration into the genome, and guaranteeing safe integration locations.

Gene Therapy Vectors

  • Adenovirus 5, AAV2, and retroviruses (lentiviruses) are examples of gene therapy vectors.
  • Vectors are crucial for delivering healthy genes for treatment.
  • Viral vectors are used because viruses naturally deliver genetic material to specific cells.
  • Gene therapy aims to deliver DNA only to the necessary cells, like those in the retina in the case of blindness, not to every cell in the body.
  • Permanent integration into a patient's DNA is vital for long-term gene therapy effectiveness.
  • Safe placement of the inserted DNA into the genome to avoid disrupting other important processes within the cell is critical.

Designer Babies: Ethical Considerations

  • The potential for genetic enhancement raises ethical concerns about creating "perfect" individuals with manipulated genes.

Current Reproductive Technologies

  • Parents can currently select the gender of their child using sperm sorting techniques and can choose embryos without certain genetic defects using preimplantation genetic diagnosis (PGD).
  • These selections already exist and could potentially be extended to other traits if sufficient resources are available.

Future Reproductive Technologies

  • Reproductive cloning, to create a copy of an individual, differs from therapeutic cloning, designed to create a copy of a body part for therapeutic purposes, like a new kidney or limb.
  • These technologies raise questions about the boundaries of medicine and the ethics of intervening in natural processes.

Key Ethical Questions

  • What are the ethical limits of genetic manipulation?
  • How far is too far in using genetic technologies for human enhancement and reproductive choices?
  • When does medical intervention become unnatural modification of life?

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Description

Explore gene therapy's potential to treat diseases by replacing faulty genes with functional ones. Various vectors, including adenoviruses and retroviruses, deliver healthy genes to target cells. Challenges remain in targeted delivery, integration, and safety.

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