Imagine, curing a fatal disease before birth. In an article form Science Daily a research shows that the gene
editing tool CRISPR is helping make this possible. Using an animal model, a
team at the Children’s Hospital of Philadelphia has managed to out play a
lethal lung disease which causes death hours after birth. The team is hoping to
solve congenital diseases like cystic fibrosis. Using precisely timed in utero
delivery of the CRISPR reagents into the amniotic fluid during the fetal
development they were able to target changes in the lungs of mice. They
introduced the gene editors four days before birth, which would be the third
trimester for humans. In a second experiment they were able to reduce the
severity of a lung disease known as surfactant protein C deficiency or SFTPC,
which has a common disease causing mutation in the human SFTPC gene. 100% of
the untreated mice died of respiratory failure within hours of birth. However,
the treated mice showed a 22% survival rate. When I think of genetics the
progress we’ve made is barely scratching the surface of the possibilities that
lie ahead. Imagine a world where we don’t have to fear for the health of our
unborn children. Through genetics, a disease free world doesn’t seem like such
a far fetched idea.
