Strategies for treating genetic disorders

• What are the different types of chromosomal aberrations?

  • Structural chromosomal defects
  • Numerical chromosomal defects
• What are genetic disorders caused by?

  Problems with DNA
• What is a polygenic disorder?

  A disorder influenced by multiple genes
• What are chromosomal aberrations?

  Structural or numerical defects in chromosomes
• What are mutations in the context of genetic disorders?

  Changes in the DNA sequence
• What are genetic disorders caused by?

  Gene mutations or chromosomal aberrations
• What are numerical defects in chromosomal aberrations?

  Defects involving an abnormal number of chromosomes
• What is the primary goal of gene therapy?

  To replace faulty genetic material
• What role do proteins play in the body?

  They are essential for body functions
• How is a chromosomal aberration similar to a cookbook issue?

  It involves having too many or too few pages
• What are the two main categories of genetic disorders?

  Gene mutations and chromosomal aberrations
• How can a mutation in DNA affect the body?

  It can lead to improper protein production
• How does the type of genetic material integration affect the treatment of disease?

  • Integrating genetic material can lead to treated disease
  • Non-integrating genetic material can also lead to treated disease, but the effect may be temporary as the genetic material dilutes
• What is the non-integrating approach in gene therapy?

  DNA floats in the cell and dilutes over time
• What are structural defects in chromosomal aberrations?

  Defects involving the structure of chromosomes
• What is a single gene disorder?

  A disorder caused by mutations in one gene
• What are vectors in gene therapy?

  Usually viruses that carry genetic material
• What is the integrating approach in gene therapy?

  DNA merges with the cell's DNA permanently
• What are the two types of genetic material integration with a cell's DNA?

  • Integrating: Genetic material integrates with the cell's DNA and remains in the DNA, even in dividing cells
  • Non-integrating: Genetic material does not permanently integrate with a cell's DNA and dilutes if the cell divides
• Why is somatic gene therapy considered safer than germline gene therapy?

  Because changes made in somatic cells do not get passed on to future generations, unlike changes made in germline cells
• What happens when DNA is compromised?

  Proteins might not work properly
• What are the main steps involved in gene therapy?

  1. Vectors carry healthy genetic material to target cells
  2. Genetic material is delivered inside the cell
  3. Functioning DNA impacts gene expression through:

  • Integrating approach: merges with cell's DNA
  • Non-integrating approach: floats in cell
• What is the scope of somatic gene therapy?

  Localized
• What is gene therapy primarily used for?

  Genetic diseases like cystic fibrosis
• What is the term used to describe genetic material that does not permanently integrate with a cell's DNA?

  Non-integrating
• What are the key differences between integrating and non-integrating genetic material?

  Integrating:

  • Genetic material integrates with cell's DNA
  • Remains in DNA even in dividing cells

  
  

  Non-integrating:

  • Genetic material does not permanently integrate with cell's DNA
  • Dilutes if the cell divides
• What are the key advantages and disadvantages of germline gene therapy compared to somatic gene therapy?

  Advantages of germline gene therapy:

  • Can correct genetic defects in all cells, including future generations

  
  

  Disadvantages of germline gene therapy:

  • Ethical concerns about modifying the human germline
  • Changes can be passed down to offspring
• What are the different types of single gene disorders?

  • Single gene disorders
  • Polygenic disorders
• What is a potential benefit of gene therapy regarding disease prevention?

  It corrects genetic defects before they manifest
• What are the key differences between somatic and germline gene therapy?

  • Somatic targets body cells; germline targets reproductive cells
  • Somatic is localized; germline is widespread
  • Somatic is not inherited; germline is inherited
  • Somatic is less controversial; germline is more controversial
• What is gene therapy?

  A technique to treat genetic disorders
• What are the consequences of somatic gene therapy compared to germline gene therapy?

  Somatic has localized effects; germline has permanent effects
• How are genetic disorders classified according to the image?

  • Gene mutation
  • Chromosomal aberrations
  • Single gene disorders
  • Polygenic disorders
  • Structural chromosomal defects
  • Numerical chromosomal defects
• What are the key components of the CRISPR/Cas9 system?

  • Cas9 nuclease
  • sgRNA (single guide RNA)
  • 20bp target sequence
• What is the scope of germline gene therapy?

  Widespread
• What is the potential duration of gene therapy compared to traditional treatments?

  Gene therapy can be potentially permanent
• Why is open public dialogue important in gene therapy ethics?

  • Navigates complex ethical dilemmas
  • Engages diverse perspectives
  • Promotes informed decision-making
• What is the genome size of the Trait virus?

  Not provided
• What does somatic gene therapy target?

  Body cells
• Is the Trait virus enveloped?

  Enveloped