People with genetic skin diseases are frequently symptomatic since birth, and such diseases can be severe, chronic, and hard to treat.1 One example is epidermolysis bullosa, which causes the skin to blister and break, leaving gaping, slow-healing wounds.2 At this year’s American Society of Gene and Cell Therapy conference, researchers presented preliminary work suggesting that lipid nanoparticles could one day deliver mRNA-based gene therapies to treat skin diseases in utero.
By injecting lipid nanoparticles directly into the amniotic fluid of pregnant mice, the researchers successfully delivered mRNA molecules to fetal skin. Combined with gene editing machinery like CRISPR, the technology may one day lead to treatments for genetic skin diseases before babies are born.
Lipid nanoparticles are tiny shells made of positively charged lipids, which bind to the negatively charged backbone of their mRNA cargo. Pegylated lipids, phospholipids, and cholesterol all help stabilize the particle, contribute to its structure, and ...
