mRNA therapy: A novel approach for retinal neurodegenerative diseases - PubMed
4 hours ago
- #lipid nanoparticles
- #mRNA therapy
- #retinal neurodegeneration
- mRNA therapy is a promising alternative to traditional gene therapies for retinal neurodegenerative diseases.
- Current gene therapies face limitations like safety concerns, limited efficacy, and cargo size restrictions.
- mRNA therapy allows rapid and efficient protein expression without genomic integration risks.
- Recent advances include engineered mRNA formats (chemically modified linear, circular, and self-amplifying RNA) for higher translation efficiency.
- Nanocarriers, especially lipid nanoparticles, improve mRNA stability, transfection efficiency, and targeted delivery to retinal cells.
- Preclinical studies show mRNA therapy's potential in neuroprotection, axon regeneration, and neurovascular regulation.
- Despite preclinical success, mRNA therapy has not yet entered clinical trials for retinal diseases.
- The review highlights the need for tailored mRNA strategies for complex retinal disorders.
- mRNA therapy supports multitarget, repeatable, and stage-specific interventions for dynamic disease progression.