A
40-year-old individual with well-controlled HIV enrols in a study testing a
genome-editing approach to reduce viral susceptibility by disrupting the CCR5
co-receptor in their CD4+ T cells. Investigators must choose a delivery method
that maximizes editing efficiency while minimizing systemic exposure and
insertional risks.

Which
delivery approach BEST aligns with the established strategy for CCR5-directed
ZFN therapies?

Question

A
40-year-old individual with well-controlled HIV enrols in a study testing a
genome-editing approach to reduce viral susceptibility by disrupting the CCR5
co-receptor in their CD4+ T cells. Investigators must choose a delivery method
that maximizes editing efficiency while minimizing systemic exposure and
insertional risks.

Which
delivery approach BEST aligns with the established strategy for CCR5-directed
ZFN therapies?

Accepted Answer

Ex
vivo gene editing of the patient’s T cells with ZFNs, followed by reinfusion of
the cells

Answer

Intravenous
administration of a viral vector encoding ZFNs for direct transduction of T
cells in the body

Answer

In
vivo editing of circulating T cells with ZFNs, then harvesting and reinfusing
the edited cells

Answer

Systemic
infusion of purified ZFN proteins to allow direct uptake by circulating T cells

Crowdly

A 40-year-old individual with well-controlled HIV enrols in a study testing a g...

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