Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery.

ABSTRACT: Congenital disorders resulting in pathological protein deficiencies are most often treated postnatally with protein or enzyme replacement therapies. However, treatment of these disorders in utero before irreversible disease onset could significantly minimize disease burden, morbidity, and mortality. One possible strategy for the prenatal treatment of congenital disorders is in utero delivery of messenger RNA (mRNA). mRNA is a nucleic acid therapeutic that has previously been investigated as a platform for protein replacement therapies and gene editing technologies. While viral vectors have been explored to induce intracellular expression of mRNA, they are limited in their clinical application due to risks associated with immunogenicity and genomic integration. As an alternative to viral ve

SUBMITTER: Swingle KL 

PROVIDER: S-EPMC8776620 | biostudies-literature | 2022 Jan

REPOSITORIES: biostudies-literature