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A long-term follow-up study of patients treated with a viral vector–based thera...

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A long-term follow-up study of patients treated with a viral

vector–based therapy for a genetic brain disorder shows stable expression of

the therapeutic protein years after treatment. Importantly, there is no

evidence of insertional mutagenesis, abnormal cell proliferation, or disruption

of endogenous gene regulation.

Which feature of the viral vector most plausibly explains

both the durability and safety of this therapeutic outcome?

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