Lipid nanoparticle (LNP)–based mRNA delivery holds promise for the treatment of inherited
retinal degenerations. Currently, LNP-mediated mRNA delivery is restricted to the retinal …

Retinal gene therapy has had unprecedented success in generating treatments that can halt
vision loss. However, immunogenic response and long-term toxicity with the use of viral …

Ocular delivery of lipid nanoparticle (LNPs) packaged mRNA can enable efficient gene
delivery and editing. We generated LNP variants through the inclusion of positively charged …

Gene therapy is now an effective approach to treat many forms of retinal degeneration.
Delivery agents that are cell-specific, allow for multiple dosing regimens, and have low …

Application of viruses as a carrier, though not safe, to deliver genes to eye tissue was
successful. However, a safer, nonviral, biocompatible lipid-based nanoparticle has never …

Lipids contain hydrocarbons and are the building blocks of cells. Lipids can naturally form
themselves into nano-films and nano-structures, micelles, reverse micelles, and liposomes …

Lipid nanoparticle (LNP)–mRNA complexes are transforming medicine. However, the
medical applications of LNPs are limited by their low endosomal disruption rates, high …

Lipid nanoparticle (LNP)-mediated nucleic acid therapies, including mRNA protein
replacement and gene editing therapies, hold great potential in treating neurological …

Delivery of mRNA-based therapeutics to the perinatal brain holds great potential in treating
congenital brain diseases. However, nonviral delivery platforms that facilitate nucleic acid …

Lipid nanoparticle (LNP)-based drug delivery systems have become the most clinically
advanced non-viral delivery technology. LNPs can encapsulate and deliver a wide variety of …