A paediatric
patient is enrolled in an experimental gene therapy trial for a genetic disease
caused by loss of function in a very large protein. Because the full-length
coding sequence exceeds the packaging capacity of adeno-associated virus (AAV)
vectors, researchers divide the gene into two separate vectors and incorporate
intein sequences to restore protein function after delivery.

What
makes inteins especially valuable for this gene therapy strategy?

Question

A paediatric
patient is enrolled in an experimental gene therapy trial for a genetic disease
caused by loss of function in a very large protein. Because the full-length
coding sequence exceeds the packaging capacity of adeno-associated virus (AAV)
vectors, researchers divide the gene into two separate vectors and incorporate
intein sequences to restore protein function after delivery.

What
makes inteins especially valuable for this gene therapy strategy?

Accepted Answer

They
permit large proteins to be reconstructed inside the cell from separately
delivered gene fragments

Answer

They
enable multiple therapeutic genes to be packaged together within a single AAV
vector

Answer

They
increase the maximum DNA payload that can be physically packaged into an AAV
capsid

Answer

They
allow oversized genes to be delivered intact by modifying the AAV genome

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A paediatric patient is enrolled in an experimental gene therapy trial for a ge...

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