Ethical considerations

• What is the significance of fixing or replacing faulty genes in gene therapy?

  It targets the underlying cause of genetic diseases
• How does gene therapy improve quality of life?

  By addressing the root cause of the disease
• What are unintended consequences of gene therapy?

  Unexpected health problems may arise
• Why do scientists and doctors prioritize safety in gene therapy?

  To prevent harmful side effects and health issues
• What is the purpose of careful gene delivery in gene therapy?

  To use safe carriers for gene delivery
• What is another concern regarding viral vectors in gene therapy?

  They might insert into harmful DNA locations
• What is the overall potential of gene therapy despite its risks?

  It offers great potential for treatment
• Why might delivered genes cause side effects?

  If they affect other genes or body reactions
• What are viral vectors used for in gene therapy?

  To carry new genes
• What are the main components of gene therapy?

  • Replacing faulty genes
  • Modifying existing genes
  • Treating genetic diseases
  • Aiming for cures of disorders
• What are researchers exploring to improve gene therapy safety?

  • Safer delivery methods like non-viral vectors
  • Refining gene targeting techniques
• What is a concern associated with viral vectors in gene therapy?

  They might cause an immune response
• What does precise targeting ensure in gene therapy?

  That the new gene goes where needed
• How can gene therapy help people with cystic fibrosis?

  By correcting the faulty gene causing mucus buildup
• How do regulations in gene therapy help patients?

  They maximize benefits while minimizing risks
• Does somatic cell gene therapy affect future generations?

  No, it only treats the individual
• What are the potential benefits of germline gene therapy?

  It could eradicate genetic diseases from family lines
• Why is it important to understand the risks of gene therapy?

  To make informed decisions about treatment
• What is a potential risk of gene therapy?

  Side effects from delivered genes
• What does the expense of gene therapy create in healthcare?

  It creates inequity
• What are the key safety measures in gene therapy?

  • Careful Gene Delivery
  • Precise Targeting
  • Ongoing Monitoring
• What is somatic cell gene therapy?

  It targets genes in specific body cells
• What is gene therapy?

  Replacing or modifying faulty genes
• How does gene therapy relate to computer programs?

  It's like updating faulty code in a program
• What systems are needed to improve access to gene therapy?

  • Ensure availability to all who need it
  • Address economic status disparities
  • Overcome geographic location barriers
• How can gene therapy affect future generations?

  • Germline gene therapy: changes genes in sperm or egg cells
  • Somatic cell gene therapy: targets specific body cells
• Why is ongoing monitoring important after gene therapy treatment?

  To check for any side effects
• What are the main responsibilities of the MHRA regarding gene therapy?

  • Approval processes for therapies
  • Safety monitoring for adverse effects
  • Ethical oversight for patient protection
• How does continuous research contribute to gene therapy?

  It helps improve safety and viability
• What is germline gene therapy?

  It changes genes in sperm or egg cells
• What must gene therapies undergo before approval?

  Rigorous testing and trials
• What ethical concerns are associated with germline gene therapy?

  Unintended effects on future generations
• What role does regulatory oversight play in gene therapy?

  It helps ensure safety improvements
• How could a new gene intended to fix muscle weakness lead to complications?

  It could trigger an immune response
• Why might some people have access to gene therapy treatments?

  They live in countries with strong healthcare systems
• If gene therapy for cystic fibrosis costs $£500,000$, who can typically afford it?


  Only the wealthiest families
• What is gene therapy used for?

  Treating or curing genetic diseases
• What are the main risks associated with gene therapy?

  • Side effects from delivered genes
  • Unintended consequences leading to health problems
  • Potential immune responses to new genes
• What is gene therapy?

  A technique to modify genes for treatment
• What is a significant drawback of gene therapy?

  It is expensive