Mutations in RPE65 impair the visual cycle in retinal cells,
leading to early-onset vision loss. An AAV-mediated gene therapy delivers a
functional copy of RPE65 to retinal tissue. This results in in sustained
protein expression and lasting visual improvement following a single administration;
even though no permanent changes are made to the patient’s genome.

Which characteristic of the AAV delivery system most
directly accounts for this durable therapeutic benefit?

Question

Mutations in RPE65 impair the visual cycle in retinal cells,
leading to early-onset vision loss. An AAV-mediated gene therapy delivers a
functional copy of RPE65 to retinal tissue. This results in in sustained
protein expression and lasting visual improvement following a single administration;
even though no permanent changes are made to the patient’s genome.

Which characteristic of the AAV delivery system most
directly accounts for this durable therapeutic benefit?

Accepted Answer

Long-term
maintenance of the introduced RPE65 gene as non-integrated episomal DNA within
the nucleus

Answer

Repair
of the mutant RPE65 locus through error-prone end-joining mechanisms

Answer

Stable
integration of the therapeutic RPE65 sequence into the host genome as an
episomal element

Answer

Precise
replacement of the mutant RPE65 allele through homologous recombination

Crowdly

Mutations in RPE65 impair the visual cycle in retinal cells, leading to early-o...

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