Self-amplifying RNA therapy encoding CNTF with disulfiram co-delivery promotes optic nerve repair through microglial pyroptosis inhibition and RGC axonal regeneration
In summary, our results demonstrate that a coordinated strategy targeting both neuroinflammatory mechanisms and regenerative pathways yields superior therapeutic outcomes in TON. This work underscores the potential of integrated RNA-small molecule therapies as a promising multi-target treatment paradigm, with broad applicability for other neuroinflammatory and neurodegenerative diseases.
Authors
- Qin Wei (ORCID: https://orcid.org/0009-0008-5942-7718)
- Qianyue Zhang (ORCID: https://orcid.org/0009-0002-5504-0301)
- Yusha Liu (ORCID: https://orcid.org/0000-0001-9253-9916)
- Siwei Liu (ORCID: https://orcid.org/0000-0001-7060-8167)
- Huifang Zhou (ORCID: https://orcid.org/0009-0004-2199-3854)
- Xianqun Fan (ORCID: https://orcid.org/0000-0001-7442-1391)
- Yingzhi Chen
- Rui Huang
- Kexin Tan
- Gang Du
- Jipeng Li
- Tingyu Deng
- Jun Zhang
- Ping Gu
- Chutong Zhang
- Ni Ni
- Mingyang Song
- Haiyang Zhang
Institutions
- Boston Children's Hospital (US)
- Harvard University (US)
- Shanghai Ninth People's Hospital (CN)
Publication Details
- Journal
- Journal of Nanobiotechnology
- Published
- 2026-03-13
- DOI
- https://doi.org/10.1186/s12951-026-04272-x
- Primary Topic
- Inflammasome and immune disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00