Podcast
Questions and Answers
What role does ornithine transcarbamylase (OTC) play in the body?
What role does ornithine transcarbamylase (OTC) play in the body?
How does the immune system respond to cells expressing a transgene from an adenovirus?
How does the immune system respond to cells expressing a transgene from an adenovirus?
What is a potential future application of gene therapy mentioned in the context?
What is a potential future application of gene therapy mentioned in the context?
What is the main function of the proposed glasses that process visual information?
What is the main function of the proposed glasses that process visual information?
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What happens to retinal ganglion cells when optogenetic components are successfully delivered?
What happens to retinal ganglion cells when optogenetic components are successfully delivered?
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What is one of the main advantages of ex vivo gene therapy?
What is one of the main advantages of ex vivo gene therapy?
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Which factor affects transgene expression in gene therapy?
Which factor affects transgene expression in gene therapy?
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What role do tissue-specific promoters play in gene therapy?
What role do tissue-specific promoters play in gene therapy?
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What is a potential consequence of an immune response to the transgene?
What is a potential consequence of an immune response to the transgene?
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What is one drawback of direct injection of vectors into blood or tissue?
What is one drawback of direct injection of vectors into blood or tissue?
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Study Notes
Gene Therapy: Challenges and Future Directions
- Targeting Specific Cells: Herpes viruses only affect certain cells. Viral vectors used in gene therapy must be carefully modified before use. Methods like electroporation, direct injection, and particle bombardment have limitations. Liposomes are another delivery option. CRISPR technology presents potential for future advancements in targeted gene editing.
Transgene Expression and Localization
- Ensuring proper transgene expression: The transgene must be expressed in the correct cells at a sufficient level and for an appropriate duration. Ex vivo gene therapy (removing and editing cells) improves targeting. Strong promoters are used to ensure high expression levels. Tissue-specific promoters can localize expression. Insulators can prevent inappropriate transgene expression due to integration near genes not meant to be affected.
Immune Response to Gene Therapy
- Immune response consequences: Immune reactions to viral vectors or transgene products can reduce transgene expression or cause lethal immune responses. Examples like adenovirus (common cold virus) can trigger immune responses against both the virus and the transgene. Cells expressing the transgene and viral components are destroyed by the immune system. An example of the potential lethality is seen in OTC deficiency.
Future Directions: Gene Therapy Applications
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Retinal Ganglion Cell Targeting: Gene therapy for lost photoreceptor function is developing. Therapies target retinal ganglion cells (output cells) to restore vision. Viral delivery of optogenetic components to retinal ganglion cells allow for light-activated responses.
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Retinal Prosthetics and Optogenetics: Combining viral insertion of optogenetic components with retinal ganglion cells and external devices is a promising future direction. Devices process visual information in a manner mimicking the retina and stimulate ganglion cells with light signals, which are then transmitted to the brain. This could improve the representation of normal retinal processing.
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Description
This quiz explores the challenges and future possibilities in gene therapy. It covers topics such as targeting specific cells, ensuring proper transgene expression, and the immune response to gene therapy. Discover the role of technologies like CRISPR and the importance of using appropriate delivery methods.
