Recombinant adeno-associated virus type 2-mediated gene delivery into the <i>Rpe65</i><sup>-/-</sup> knockout mouse eye results in limited rescue.

Genet Vaccines Ther · 2004 · PMC416492 · PMID 15109394

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may be significantno p-value reported

If this hypothesis is true, the implications for rAAV-mediated gene therapy as a clinical option may be significant, as it may be essential to deliver rAAV.RPE65 at the time when RPE65 expression should be commencing in order to completely compensate for its loss.

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