Tuesday, April 30, 2019

CRISPR


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.

2 comments:

  1. I agree that we are only at the tip of the iceberg when it comes to genetics. Just think of how much progress has been made in the last twenty years, and then how much more can be made in the same amount of time into the future. Maybe CRISPR will be introduced to clinical trials by then. Hopefully scientists figure out a way to eliminate the off-target gene effects of the mechanism and are able to cure genetic diseases without any negative repercussions.

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  2. I agree, we've barely scratched the surface on the capabilities of genes and gene manipulation can do. Although its terrifying to think, I feel if we find a way to boost mankind's ability to resist all infections/diseases, the poor would be exploited and only the rich may be able to afford it.

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