Gene Therapy: Challenges and Future Directions
10 Questions
0 Views

Choose a study mode

Play Quiz
Study Flashcards
Spaced Repetition
Chat to Lesson

Podcast

Play an AI-generated podcast conversation about this lesson

Questions and Answers

What role does ornithine transcarbamylase (OTC) play in the body?

  • It regulates the immune response to pathogens.
  • It synthesizes essential fatty acids for the liver.
  • It removes excess nitrogen from the bloodstream. (correct)
  • It absorbs amino acids from digested proteins.

How does the immune system respond to cells expressing a transgene from an adenovirus?

  • It stimulates increased production of the transgene.
  • It kills those cells expressing the transgene. (correct)
  • It enhances those cells' function.
  • It ignores those cells completely.

What is a potential future application of gene therapy mentioned in the context?

  • Development of vaccines for viral infections.
  • Therapies for endocrine system anomalies.
  • Creation of hormonal therapies for metabolic disorders.
  • Creation of retinal prosthetics and optogenetics. (correct)

What is the main function of the proposed glasses that process visual information?

<p>To mimic retinal functions and assist with vision. (C)</p> Signup and view all the answers

What happens to retinal ganglion cells when optogenetic components are successfully delivered?

<p>They respond to light that is projected onto them. (B)</p> Signup and view all the answers

What is one of the main advantages of ex vivo gene therapy?

<p>It ensures that only the correct cells are targeted for treatment. (D)</p> Signup and view all the answers

Which factor affects transgene expression in gene therapy?

<p>The presence of strong promoters driving expression. (A)</p> Signup and view all the answers

What role do tissue-specific promoters play in gene therapy?

<p>They localize expression to specific tissues. (B)</p> Signup and view all the answers

What is a potential consequence of an immune response to the transgene?

<p>Reduction in transgene expression. (D)</p> Signup and view all the answers

What is one drawback of direct injection of vectors into blood or tissue?

<p>It is often inefficient and dependent on the tissue type. (A)</p> Signup and view all the answers

Flashcards

Gene Therapy Vectors

Tools used to deliver genes into cells for treatment.

Transgene Expression Levels

The amount and duration of a transgene's activity in target cells.

Immune Responses to Gene Therapy

Reactions of the body's immune system to the vector or transgene products.

Tissue-Specific Promoters

Promoters ensuring gene expression only in target cells.

Signup and view all the flashcards

Correct Cell Targeting

Ensuring the gene therapy treats only the intended cells.

Signup and view all the flashcards

Adenovirus & Gene Therapy

Adenoviruses are commonly used as vectors in gene therapy. However, they can trigger strong immune responses, which might lead to cell death, making the therapy ineffective or even dangerous.

Signup and view all the flashcards

OTC Deficiency

A genetic disorder where the liver enzyme ornithine transcarbamylase (OTC) is deficient. This results in the body's inability to process excess nitrogen, which can be fatal.

Signup and view all the flashcards

Optogenetics: Restoring Sight

A gene therapy approach that uses light-sensitive proteins to activate retinal ganglion cells, restoring visual function in patients with retinal degeneration.

Signup and view all the flashcards

Retinal Prosthetics

Artificial devices designed to replace damaged photoreceptor cells in the eye. They use cameras and computer processing to convert visual information into electrical signals that stimulate retinal ganglion cells.

Signup and view all the flashcards

Optogenetic Eye Glasses

Specialized glasses that combine camera technology, computer processing, and optogenetic gene therapy to provide a visual experience closer to normal vision for people with retinal degeneration.

Signup and view all the flashcards

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

  • 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.

  • 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.

Studying That Suits You

Use AI to generate personalized quizzes and flashcards to suit your learning preferences.

Quiz Team

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.

More Like This

Use Quizgecko on...
Browser
Open
Browser
Browser