Structure · dataset · 2026
Data from: A combinatorial synthetic strategy for developing genome-editing protein-delivery agents targeting mouse retina
Listed in DataCite
CRISPR/Cas9-based gene-editing technologies offer promise for treating inherited retinal diseases (IRDs); however, safe and efficient ocular delivery of precision editors remains challenging.
Description
To address this challenge, we report a new class of Coomassie brilliant blue (CBB)-derived lipidoids that bind and deliver proteins. Subretinal injection of Cre complexed with these lipidoids into mT/mG mice leads to robust recombination in the retinal pigment epithelium and photoreceptors.
We employ the CBB-lipidoid platform to deliver adenine base editor (ABE) ribonucleoproteins (RNP). Incorporating CBB lipidoids into liposomes improves delivery efficiency. CBB11 stands out for facilitating precise in vivo ABE-mediated gene editing.
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Delivery of liposome-CBB11-RNP complexes results in a 120-fold increase in base editing compared to RNP alone and restores the scotopic ERG b-wave response in the rd12 mouse model. These results demonstrate the potential of CBB-augmented, liposome-RNP systems for therapeutic gene editing in the eye, paving the way for single-dose precision medicines to treat IRDs.
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Where it is published
- Repository landing page datadryad.org/dataset/doi:10.5061/dryad.d51c5b0hd ↗
landing page · from DataCite
- DOI doi.org/10.5061/dryad.d51c5b0hd ↗
DOI / persistent id · from DataCite
Documentation and papers
- Creative Commons Zero v1.0 Universal creativecommons.org/publicdomain/zero/1.0/legalcode ↗
license · from DataCite
- IsCitedBy 10.1038/s41467-026-69077-w doi.org/10.1038/s41467-026-69077-w ↗
publication · from DataCite
Catalogue records · 2
- DataCite API api.datacite.org/dois/10.5061/dryad.d51c5b0hd ↗
metadata API · from DataCite
- DataCite Commons commons.datacite.org/doi.org/10.5061/dryad.d51c5b0hd ↗
catalogue entry · from DataCite
Topics
Provenance · 1 source records, 15 field assertions
| Source | Key | Last seen | Raw |
|---|---|---|---|
| DataCite | 10.5061/dryad.d51c5b0hd | 7 d ago | JSON v1 |
| Field | Assertion | Extractor | Evidence |
|---|---|---|---|
| access_level | source · DataCite | connector:datacite@1.0.0 | /data/attributes/rightsList |
| byte_size | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:biological-sciences | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:chemical-sciences | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:engineering-and-technology | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:health-sciences | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:materials-engineering | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:medical-and-health-sciences | source · DataCite | connector:datacite@1.0.0 | |
| concepts[field].fos:medical-engineering | source · DataCite | connector:datacite@1.0.0 | |
| created_date | source · DataCite | connector:datacite@1.0.0 | |
| description | source · DataCite | connector:datacite@1.0.0 | /data/attributes/descriptions |
| license | source · DataCite | connector:datacite@1.0.0 | /data/attributes/rightsList |
| publication_date | source · DataCite | connector:datacite@1.0.0 | /data/attributes/dates |
| title | source · DataCite | connector:datacite@1.0.0 | /data/attributes/titles/0/title |
| version_label | source · DataCite | connector:datacite@1.0.0 |