Hugo Malki

This study compares immune responses to AAV-delivered CRISPR-Cas9 and RNP-delivered CRISPR-Cas9 in the retina, finding persistent inflammation with AAV and transient inflammation with RNP.

The paper compares CRISPR/Cas9 gene editing efficiency in retinal organoids, HEK293T cells, and mouse models, finding retinal organoids better mirror in vivo editing outcomes.

This study delivers Cas9 or base editor RNP complexes directly to retinal cells, achieving gene editing in photoreceptors and RPE, but with toxicity at high concentrations.