6.3.5 Gene therapy

• Somatic cell gene therapy targets specific body cells and does not affect future generations
• Match the type of gene therapy with its definition and impact:

  Somatic cell gene therapy ↔️ Targets specific body cells, does not affect future generations

  Germline gene therapy ↔️ Modifies reproductive cells, impacts future offspring
• What is the primary purpose of gene therapy?

  Treat or prevent diseases
• Germline gene therapy is less ethically controversial than somatic cell gene therapy.

  False
• Which type of gene therapy affects only the treated individual?

  Somatic cell gene therapy
• Somatic cell gene therapy is primarily used to treat single-gene disorders.

  True
• Non-viral vectors are considered safer because they are less immunogenic
• Which genetic disorder has shown promise in treatment using gene therapy?

  Hemophilia
• Gene therapy involves replacing a faulty gene or adding a new gene to correct a genetic defect.


  True
• What type of cells are targeted in somatic cell gene therapy?

  Body cells
• Which type of gene therapy is more controversial due to ethical concerns about inheritable genetic changes?

  Germline gene therapy
• Germline gene therapy is widely approved and used in most countries.

  False
• Viral vectors are highly efficient at delivering genes into cells
• Viral vectors in gene therapy pose a risk of insertional mutagenesis.


  True
• Which single-gene disorder is commonly treated using gene therapy to restore normal blood clotting ability?

  Hemophilia
• Gene therapy can be used to treat diseases like Alzheimer's and Parkinson's.


  True
• Germline gene therapy raises ethical questions about altering the human genome
• What is a key ethical concern about germline gene therapy?

  Inheritable genetic changes
• Germline gene therapy raises ethical concerns due to the possibility of inheritable genetic changes
• What is a key advantage of using viral vectors in gene therapy?

  Highly efficient gene delivery
• Match the genetic disease with its potential treatment using gene therapy:

  Cystic fibrosis ↔️ Replacing faulty CFTR gene

  Sickle cell disease ↔️ Correcting hemoglobin gene
• Gene therapy is a technique that introduces genetic material into cells to treat or prevent diseases
• Match the type of gene therapy with its description:

  Somatic cell gene therapy ↔️ Targets specific body cells

  Germline gene therapy ↔️ Modifies reproductive cells
• Somatic cell gene therapy affects future generations.

  False
• Germline gene therapy can alter the genetic makeup that is passed down to future generations
• What are the two main methods used in gene therapy to deliver genetic material into target cells?

  Viral and non-viral vectors
• What is a key advantage of non-viral vectors in gene therapy?

  Safer and less immunogenic
• Match the application of gene therapy with its description:

  Monogenic Disorders ↔️ Treat single-gene diseases

  Cancer ↔️ Targets cancer-causing mutations

  Other Genetic Diseases ↔️ Potential treatment for Parkinson's
• In cancer treatment, gene therapy can boost the immune system's ability to fight cancer cells
• What is one safety concern associated with using viral vectors in gene therapy?

  Insertional mutagenesis
• Steps involved in gene therapy using viral vectors

  1️⃣ Modify the virus to carry the desired gene

  2️⃣ Deliver the modified virus into target cells

  3️⃣ Virus infects the cells and releases the gene

  4️⃣ The gene is integrated into the cell's DNA