Lipid nanoparticle-mediated delivery of mRNA into the mouse and human retina and other ocular tissues.
Cheri Z ChambersGillian L SooAbbi L Engelnull nullIan A GlassAndrea FrassettoPaolo G V MartiniTimothy J CherryPublished in: bioRxiv : the preprint server for biology (2023)
Ocular gene-replacement therapies using non-viral vector methods are of interest as alternatives to adeno-associated virus (AAV) vectors. Our studies show that mRNA LNP delivery can be used to transfect retinal cells in both mouse and human tissues without inducing significant inflammation. This promising methodology could be used to transfect retinal cell lines, tissue explants, mice, or potentially as gene-replacement therapy in a clinical setting in the future.
Keyphrases
- optic nerve
- replacement therapy
- endothelial cells
- diabetic retinopathy
- optical coherence tomography
- gene expression
- induced pluripotent stem cells
- gene therapy
- induced apoptosis
- genome wide
- copy number
- oxidative stress
- smoking cessation
- cell cycle arrest
- type diabetes
- sars cov
- adipose tissue
- dna methylation
- fatty acid
- metabolic syndrome
- current status
- insulin resistance