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Questions and Answers
What is the consequence of overexpressing the transferred gene in thalassemia?
What is the consequence of overexpressing the transferred gene in thalassemia?
- New imbalance of globin chains in red blood cells (correct)
- Increased expression of normal genes
- No adverse effect on the body
- Effective treatment of the disorder
What is the ideal target cell for gene therapy?
What is the ideal target cell for gene therapy?
- Muscle cells
- Stem cells (correct)
- Nerve cells
- Red blood cells
Why is it essential to culture target cells in vitro?
Why is it essential to culture target cells in vitro?
- To test the efficacy of the gene
- To reduce the risk of graft-versus-host disease
- To study the disease mechanism
- To facilitate gene transfer (correct)
What is the minimum level of expression required to be therapeutic in some enzymopathies?
What is the minimum level of expression required to be therapeutic in some enzymopathies?
What is the ideal precedent for gene therapy?
What is the ideal precedent for gene therapy?
Why is it essential to introduce a sufficient number of recipient cells into the patient?
Why is it essential to introduce a sufficient number of recipient cells into the patient?
What is the advantage of using stem cells as target cells?
What is the advantage of using stem cells as target cells?
What is the purpose of animal studies in gene therapy?
What is the purpose of animal studies in gene therapy?
Why are large animal models often preferred over small animal models?
Why are large animal models often preferred over small animal models?
What is the significance of the organ in which the gene therapy is targeted?
What is the significance of the organ in which the gene therapy is targeted?
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Study Notes
What is Gene Therapy
- Gene therapy is the introduction of a biologically active gene into a cell to achieve a therapeutic benefit.
- The first gene therapy product was licensed in the US and Europe in 2012 for the treatment of lipoprotein lipase deficiency.
- Gene therapy has now been approved for the treatment of several more disorders.
Essential Requirements of Gene Therapy for an Inherited Disorder
- Identity of the molecular defect: the affected gene must be known.
- A functional copy of the gene: a complementary DNA (cDNA) clone of the gene or the gene itself must be available.
- An appropriate vector: the ideal vector is safe, readily made, and easily introduced into the target tissue, and it would express the gene of interest for life.
Characteristics of Vectors
- Retrovirus:
- Nontoxic to the cell
- Low number of copies of the viral DNA integrate into the host genome
- Stable
- Infects dividing or nondividing cells
- Adeno-Associated Virus (AAV):
- Infects a wide variety of dividing or nondividing cell types
- Accommodates insertion of longer genes
- Adenovirus:
- Accommodates insertion of small genes
- Elicits strong immune response (restricted to specific cell types)
Additional Essential Requirements
- Knowledge of the pathophysiologic mechanism of the disease
- Favorable risk-to-benefit ratio
- Appropriate regulatory components for the transferred gene
- An appropriate target cell: ideally, the target cell must have a long half-life or good replicative potential in vivo
- Strong evidence of efficacy and safety: cultured cell and animal studies must indicate that the vector and gene construct are both effective and safe
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