Thursday, July 20, 2023

Delivering Gene Therapies in Utero to treat genetic skin diseases

Good news! Amazing stuff! This is still rather preliminary work (I was not able to find the research paper).

"... One example is epidermolysis bullosa, which causes the skin to blister and break, leaving gaping, slow-healing wounds. ... 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. ..."

Delivering Gene Therapies in Utero  | The Scientist Magazine® By delivering mRNA to the skin of mice in utero, researchers showed a proof-of-concept for shuttling gene therapies to skin cells before birth.

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