Podcast
Questions and Answers
What are some of the vector properties to be checked and described when proposing a new gene therapy approach?
What are some of the vector properties to be checked and described when proposing a new gene therapy approach?
- Transduction efficiency, targeting, and immunogenicity
- Safety, transduction efficiency, and production
- Duration of the GT effect, targeting, and immunogenicity
- Stability, expression level, and targeting (correct)
What is the main difference between IN VIVO and EX VIVO gene therapy delivery methods?
What is the main difference between IN VIVO and EX VIVO gene therapy delivery methods?
- IN VIVO involves injecting the GT directly inside the cells, while EX VIVO involves retrieving cells from the patient (correct)
- IN VIVO involves reaching the final organ directly, while EX VIVO involves retrieving cells from the patient
- IN VIVO involves selecting transduced cells in vitro, while EX VIVO involves reaching the final organ directly
- IN VIVO involves retrieving cells from the patient, while EX VIVO involves injecting the GT directly inside the cells
What does EX VIVO gene therapy delivery method involve?
What does EX VIVO gene therapy delivery method involve?
- Selecting transduced cells in vitro and injecting them back into the patient
- Retrieving cells from the patient, transducing them in vitro, and selecting the transduced cells (correct)
- Reaching the final organ directly by injecting the GT
- Injecting the GT directly inside the cells
Which of the following is NOT considered one of the vector properties for gene therapy approval?
Which of the following is NOT considered one of the vector properties for gene therapy approval?
What does targeting refer to in the context of vector properties for gene therapy?
What does targeting refer to in the context of vector properties for gene therapy?
In terms of vector properties for gene therapy, what does transduction efficiency represent?
In terms of vector properties for gene therapy, what does transduction efficiency represent?
What is an important consideration for in vivo delivery of gene therapy?
What is an important consideration for in vivo delivery of gene therapy?
Which type of injection is used when the goal is to reach as many cells as possible in gene therapy?
Which type of injection is used when the goal is to reach as many cells as possible in gene therapy?
What is a challenge for the vector in systemic injection (intravenous) in gene therapy?
What is a challenge for the vector in systemic injection (intravenous) in gene therapy?
Which barrier does the vector need to pass through in order to reach target cells in the brain?
Which barrier does the vector need to pass through in order to reach target cells in the brain?
What is the best way to deliver some proteins produced by connective tissue or dendritic cells?
What is the best way to deliver some proteins produced by connective tissue or dendritic cells?
What type of injection route can be used for treating cardiac diseases with gene therapy?
What type of injection route can be used for treating cardiac diseases with gene therapy?
Which factor needs to be considered for in vivo gene therapy targeting metastatic cells in cancer?
Which factor needs to be considered for in vivo gene therapy targeting metastatic cells in cancer?
What type of barrier does the vector need to pass through if the target cells are immune-privileged sites?
What type of barrier does the vector need to pass through if the target cells are immune-privileged sites?
Which delivery route can be used if the target tissue is easily accessible from the outside, like muscles?
Which delivery route can be used if the target tissue is easily accessible from the outside, like muscles?
What challenge is associated with vector dilution in the body fluids during intravenous injection?
What challenge is associated with vector dilution in the body fluids during intravenous injection?
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