A 7-year-old patient is diagnosed with a rare inherited
disorder caused by mutations in a gene too large to fit into a single
adeno-associated virus (AAV) vector. Researchers are designing a gene therapy
approach that splits the therapeutic gene into multiple segments and relies on
inteins to reassemble the full-length protein inside target cells.

Why are inteins particularly useful in this therapeutic
context?

Question

A 7-year-old patient is diagnosed with a rare inherited
disorder caused by mutations in a gene too large to fit into a single
adeno-associated virus (AAV) vector. Researchers are designing a gene therapy
approach that splits the therapeutic gene into multiple segments and relies on
inteins to reassemble the full-length protein inside target cells.

Why are inteins particularly useful in this therapeutic
context?

Accepted Answer

They
overcome the size limitation of therapeutic genes used in AAV-mediated gene
therapy by enabling in-cell reconstitution

Answer

They
allow larger than average gene constructs to be encapsulated by a single AAV
vector

Answer

They
allow multiple smaller gene constructs to be packaged simultaneously in a
single AAV vector

Answer

They
allow smaller than average gene constructs to be encapsulated by a single AAV
vector

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A 7-year-old patient is diagnosed with a rare inherited disorder caused by muta...

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