16 Questions
What are some of the vector properties to be checked and described when proposing a new gene therapy approach?
Stability, expression level, and targeting
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
What does EX VIVO gene therapy delivery method involve?
Retrieving cells from the patient, transducing them in vitro, and selecting the transduced cells
Which of the following is NOT considered one of the vector properties for gene therapy approval?
Type of effort required by pharmaceutical companies
What does targeting refer to in the context of vector properties for gene therapy?
How to reach the desired cells
In terms of vector properties for gene therapy, what does transduction efficiency represent?
How efficient the GT is in reaching and affecting the target cells
What is an important consideration for in vivo delivery of gene therapy?
Transduction efficiency
Which type of injection is used when the goal is to reach as many cells as possible in gene therapy?
Intravenous injection
What is a challenge for the vector in systemic injection (intravenous) in gene therapy?
Survival in blood serum
Which barrier does the vector need to pass through in order to reach target cells in the brain?
Endothelium of vessels
What is the best way to deliver some proteins produced by connective tissue or dendritic cells?
Subcutaneous injection
What type of injection route can be used for treating cardiac diseases with gene therapy?
Inhalation
Which factor needs to be considered for in vivo gene therapy targeting metastatic cells in cancer?
Transduction efficiency
What type of barrier does the vector need to pass through if the target cells are immune-privileged sites?
Endothelium of vessels
Which delivery route can be used if the target tissue is easily accessible from the outside, like muscles?
Local injection
What challenge is associated with vector dilution in the body fluids during intravenous injection?
Dilution effects of the vector in the system
Test your knowledge of vector properties in gene therapy with this quiz. Explore the considerations for vectors' effects, including safety, transduction efficiency, stability, duration of effect, and expression level.
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