Abstract
In a recent article in Molecular Therapy – Nucleic Acids,1 Sun et al. describe a promising therapy for ABCA4-mediated Stargardt disease (STGD). The therapy is based on a non-viral gene delivery platform designed for the treatment of inherited retinal diseases (IRDs) caused by mutations in large genes. This platform allows for the formulation of self-assembling multifunctional lipid nanoparticles loaded with plasmid DNA containing the gene of interest. The authors’ preliminary study using subretinal injections showed that the therapy was safe and effective for short-term gene expression in STGD1 mice.
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