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.

bioRxiv · Dec 14, 2025 · PreprintBioengineering

Retinal organoids mirror CRISPR/Cas9 gene editing efficiency observed in vivo

Juliette Pulman, Hugo Malki, Paul Oudin, Ecem Aydin, Sophie Tran, Laura Visticot, Camille Robert, Anne De Cian, Marie As, Olivier Goureau, Jean‐Paul Concordet, Deniz Dalkara

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.

Molecular Therapy — Methods & Clinical Development · Nov 11, 2025Peer-reviewedBioengineering

Inter-individual variability in immune responses to AAV-mediated ocular gene delivery across species impedes reliable immunomonitoring profile

Duohao Ren, Gaëlle Anne Chauveau, Julie Vendomèle, Emilie Cabon, Audrey Pineiro, Catherine Vignal‐Clermont, Hanadi Saliba, Giuseppe Ronzitti, Anne H. M. Galy, Deniz Dalkara, Juliette Pulman, Divya Ail, Sylvain Fisson

The paper shows that immune responses to AAV-mediated ocular gene delivery vary across species and individuals, complicating reliable immunomonitoring.

bioRxiv · Jun 2, 2025 · PreprintImmunology