Leber congenital amaurosis caused by RPE65 deficiency leads
to progressive retinal dystrophy due to impaired regeneration of 11-cis-retinal
in photoreceptor cells. Luxturna is an AAV-based gene therapy designed to
restore visual function in affected patients.

Clinical studies show sustained RPE65 expression and
improved vision for years after a single subretinal injection, despite the
absence of targeted genome editing.

Which property of the Luxturna AAV vector best explains this
long-term therapeutic effect?

Question

Leber congenital amaurosis caused by RPE65 deficiency leads
to progressive retinal dystrophy due to impaired regeneration of 11-cis-retinal
in photoreceptor cells. Luxturna is an AAV-based gene therapy designed to
restore visual function in affected patients.

Clinical studies show sustained RPE65 expression and
improved vision for years after a single subretinal injection, despite the
absence of targeted genome editing.

Which property of the Luxturna AAV vector best explains this
long-term therapeutic effect?

Accepted Answer

Persistence
of the therapeutic RPE65 transgene as an episomal DNA element in the nucleus

Answer

Correction
of the defective RPE65 gene through non-homologous end joining

Answer

Persistence
of the therapeutic RPE65 transgene as an episomal DNA element which is
integrated into the genome

Answer

Correction
of the defective RPE65 gene through homologous recombination

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