Clinical advances enable the prenatal diagnosis of genetic diseases that are candidates for
gene and enzyme therapies such as messenger RNA (mRNA)–mediated protein …

Congenital disorders resulting in pathological protein deficiencies are most often treated
postnatally with protein or enzyme replacement therapies. However, treatment of these …

Ionizable lipid nanoparticles (LNPs) are the most clinically advanced nonviral platform for
mRNA delivery. While they have been explored for applications including vaccines and …

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 …

Conspectus Since the US Food and Drug Administration (FDA) granted emergency use
authorization for two mRNA vaccines against SARS-CoV-2, mRNA-based technology has …

Treating pregnancy-related disorders is exceptionally challenging because the threat of
maternal and/or fetal toxicity discourages the use of existing medications and hinders new …

Lipid nanoparticles hold great potential as an effective non-viral vector for nucleic acid-
based gene therapy. Plasmid DNA delivery can result in extended transgene expression …

Messenger RNA (mRNA) has recently emerged as a promising class of nucleic acid therapy,
with the potential to induce protein production to treat and prevent a range of diseases …

As mRNA is degraded by nucleases and cannot easily cross the cell membrane due to its
large size and negative charge, delivery requires encapsulation in vehicles, such as lipid …

A central goal of chemical and drug delivery sciences is to maximize the therapeutic efficacy
of a given drug at the lowest possible dose. Here, we report a generalizable strategy that …