Questions and Answers
What is the primary function of hemoglobin within red blood cells?
To transport oxygen throughout the circulatory system
What type of mutation would likely impair oxygen transport in red blood cells?
Deleterious mutation
What is the goal of gene therapy in treating genetic mutations?
To introduce a functional gene to replace a mutant gene
Why is gene therapy in bone marrow stem cells the most effective treatment for the patient?
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What is the outcome of expression of the mutant hemoglobin gene?
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What is the vector used to insert functional gene copies into stem cells in gene therapy?
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Study Notes
Hemoglobin Function
- Hemoglobin is an oxygen-binding protein found in red blood cells (RBCs)
- Oxygen diffuses into the bloodstream through gas exchange in the lungs and binds to hemoglobin for transportation to tissues throughout the body
Genetic Mutations Affecting Hemoglobin
- Deleterious mutations in hemoglobin genes can impair oxygen transport
- Gene therapy can be used to treat genetic mutations by introducing a functional gene to replace a mutant gene
Gene Therapy for Hemoglobin Mutations
- A common gene therapy method uses retroviral vectors to insert functional gene copies into stem cells of patients with specific genetic mutations
- For patients homozygous for a deleterious mutation in a hemoglobin subunit gene, gene therapy can introduce and activate a functional hemoglobin gene in bone marrow stem cells
- This leads to the production of RBCs with hemoglobin that has normal oxygen-binding abilities
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Description
Learn about hemoglobin's role in oxygen transport, its genetic basis, and the impact of mutations on its function. Discover how gene therapy can be used to treat genetic mutations.
