Samantha DiGrande writing for AJMC reports researchers from Penn Medicine and Children’s Hospital of Philadelphia (CHOP) completed a successful animal model proof-of-concept with the results recently published in Science Translational Medicine. The Philadelphia-based team was able to edit out a lethal lung disease using CRISPR utero mice models.
Edward E. Morrisey, PhD and professor of Cardiovascular Medicine and Cell and Developmental Biology in the Perelman School of Medicine at the University of Pennsylvania reported “we wanted to know if this could work at all. The trick was how to direct the gene-editing machinery to target cells that line the airways of the lungs.”
DiGrande reports that the translational research team is in pursuit of identifying CRISPR editing as a means of solving condition such as congenital disease like cystic fibrosis, surfactant protein deficiency, and alpha-1 antitrypsin. AMJC notes the researchers reveals that with precise delivery of the in utero delivery of CRISPR gene-editing reagents to the amniotic fluid resulted in targeted changes in the mice’ lungs. This represents what TrialSite News is observing to be an intensification of CRISPR related experiments. This team for example was able to “deactivate the mutation responsible for surfactant protein C (SFTPC) deficiency, and more than 22% of the mice survived. If left untreated, 100% of mice die from respiratory failure hours after birth.”