A patient receives an experimental gene therapy to treat an
inherited neurological disorder. Several years later, follow-up imaging and
biopsies show persistent expression of the therapeutic protein, with no
evidence of oncogenic transformation or disruption of host gene function in
treated cells.

Which property of the viral vector used in this therapy most
likely contributed to this favourable long-term safety outcome?

Question

A patient receives an experimental gene therapy to treat an
inherited neurological disorder. Several years later, follow-up imaging and
biopsies show persistent expression of the therapeutic protein, with no
evidence of oncogenic transformation or disruption of host gene function in
treated cells.

Which property of the viral vector used in this therapy most
likely contributed to this favourable long-term safety outcome?

Accepted Answer

Its
very low frequency of random integration, allowing the transgene to persist
mainly as episomal DNA

Answer

Its
ability to selectively integrate the therapeutic DNA into genomic “safe
harbour” sites

Answer

Its
tendency to elicit weak immune responses that avoid elimination of transduced
cells

Answer

Its
tendency to elicit only mild immune responses that avoid elimination of
transduced cells

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A patient receives an experimental gene therapy to treat an inherited neurologi...

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