Studies and research continue to hit new milestones through CRISPR. CRISPR is a gene editing technique that, according to scientists, can cure and prevent countless diseases and disorders. Already, this tool has seen widespread success as well as controversy for its future implications in genetic manipulation. At the at Children’s Hospital of Philadelphia (CHOP), researchers have recently used CRISPR to prevent a deadly metabolic disorder in prenatal lab mice. Even though this study is still in the early stages and more research needs to be done, these same techniques can potentially be used on humans to prevent congenital diseases even before birth.
By using CRISPR-Cas9 and base editor 3 (BE3) gene editing, researchers were able to cut cholesterol levels in healthy mice treated in utero. They were able to do this by targeting a gene that regulates those levels and prenatal gene editing that improved liver function and prevented neonatal death. Scientists were able to successfully engineer the mice with a mutation that caused the lethal liver disease hereditary tyrosinemia type 1 (HT1). This means a lot for humans because HT1 is frequently found in infants. If initial treatment for HT1 fails, it can likely lead to cancer and liver failure. Addressing these diseases before they can affect quality of life is important for young patients.
The treatment is both simple and incredibly complex. The research procedure involves opening the uterus of a pregnant mouse, removing the fetus, and injecting CRISPR into the vitelline vein which connects to the liver. This ensures the CRISPR is going directly into the liver and nowhere else, so no other organs were affected. The mice involved in this research also did not show any side effects to the treatment. Precise targeting is part of what makes CRISPR such an enticing prospect for the medical field at large.
According to William H. Peranteau, the study’s co-leader and a pediatric and fetal surgeon in CHOP’s Center for Fetal Diagnosis and Treatment, the ultimate goal through this study is to intervene the disease and be able to treat it prenatally. These proof-of-concept studies will require a lot more research and testing of lab animals but show a lot of promise. If all goes well, human infants will no longer have to suffer from liver diseases.