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TL;DR
It found that an anionic LNP surface charge led to successful photoreceptor transfection, but the specific LNPs carrying Cas9 and sgRNA cargo did not achieve this delivery.
Nature Communications · 9 authors, 3 centres
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It found that an anionic LNP surface charge led to successful photoreceptor transfection, but the specific LNPs carrying Cas9 and sgRNA cargo did not achieve this delivery.
However, when the cargo was changed to Cas9 mRNA and a guide RNA (sgAi9) targeting the Ai9 reporter in a separate mouse model, the LNPs showed altered structure and a neutral surface charge, resulting in a near-complete loss of photoreceptor transfection. The work concludes that while an anionic surface charge is key for retinal delivery, further optimization of LNP physicochemical properties is needed for effective gene-editing cargo delivery. Limitations include the use of two different mouse models with varying sensitivity and the observation that the therapeutic LNP formulation had an abnormal structure.