Scientists at Penn Medicine have discovered a method of using prenatal gene editing to prevent a deathly metabolic disorder in animals. Penn Medicine News reported that this could potentially lead to a neonatal treatment for human congenital diseases down the road, opening new doors for the medical and scientific communities.
The Perelman School of Medicine and the Children's Hospital of Philadelphia conducted the study, originally published in Nature Medicine during October.
The research team used several gene-editing tools, including CRISPR-Cas 9, to influence a gene that regulates cholesterol levels. CRISPR-Cas9 is a gene editing method that was “adapted from a naturally occurring genome editing system in bacteria” and allows genetic information to be added or removed from the genome, according to Genetics Home Reference.
Thanks to these methods, the team was also able to prevent death prior to birth and improve liver function in many of these mice, another significant benefit to these new findings.
Penn researchers involved in the study included study co-leaders William H. Peranteau and Kiran Musunuru. Perenteau, a pediatric and fetal surgeon at CHOP, said to Penn Medicine that they hope to use their findings to “treat severe diseases diagnosed early in pregnancy” and to “intervene prenatally in congenital diseases that currently have no effective treatment for most patients, result in death or severe complications in infants."
These findings could have substantial implications, not only for infants suffering from congenital diseases, but also for the field of genetics in general. If scientists are able to target and eliminate diseases prior to birth, lives could be saved, and the methods could be expanded to target other diseases as well.
The University’s position at the forefront of this discovery could also mean more opportunities for research in prenatal gene editing and the development of entirely new gene-targeting methods.
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