Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target <i>ABCA4</i> splice variants in Stargardt disease.

Mol Ther Nucleic Acids · 2026 · PMC12795662 · PMID 41536809

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did not reach statistical significanceno p-value reported

However, the decrease did not reach statistical significance.

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showed a trendno p-value reported

Quantification of the disease transcript/FL expression level showed a trend of decrease for both disease transcripts in ABCA4 sgRNAs-treated group compared to the control (ΔE39/FL = 0.38-fold, ΔE39-40/FL = 0.28-fold compared to control, Figure 1 F), indicating a decrease in the expression of disease transcripts while the expression of FL transcript is maintained.

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Quoted from the open-access full text in Europe PMC under the licence the publisher applied. The sentence is reproduced exactly as published; the emphasis is ours.

Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target &lt;i&gt;ABCA4&lt;/i&gt; splice variants in Stargardt disease.

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Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target <i>ABCA4</i> splice variants in Stargardt disease.