Dacey Presnell

Human Physiology & Zoology

Ms. Sasnett

18 August 2023

Delivering Gene Therapies in Utero

Genetic diseases are frequently symptomatic from birth and difficult to treat. Through

experimentations on pregnant mice, though, researchers were able to find a possible solution to

this issue. In the article discussing this topic, the author wrote, “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.” (Mesa, Natalia. “Delivering Gene Therapies in Utero.” The Scientist, 18 July 2023.) This

statement meant that these researchers were able to find a method that could help rid genetic

diseases in utero. Through their research, they were able to successfully deliver mRNA

molecules to fetal skin. The lipid nanoparticles containing mRNA created a huge difference for

the pregnant mice and the data showed how persistent it worked as the mice aged. This evidence

indicated that the researchers had successfully targeted progenitor cells within the skin.

With this knowledge, they hope to create treatments for genetic skin diseases before babies are

even born.

This topic is not only relevant to human physiology but also to zoology. Through the

experiments mentioned in the article, researchers were able to understand and alter the genes of

pregnant mice. Most genetic diseases humans receive from their ancestors have always been

difficult to treat or incurable. With new evidence obtained through various experiments, the

likelihood of scientists implementing gene therapy may continue to grow with more information.
This research allowed researchers to learn more about the development of genetic diseases in

human bodies as well as mice, hopefully helping to reach the goal of a cure or treatment.

https://www.the-scientist.com/news-opinion/delivering-gene-therapies-in-utero-71220