Mice Gene-Edited While Still in the Womb

Link to article: mice-gene-edited-while-still-in-the-womb-65767
Supporting article: crispr-editing-out-a-deadly-lung-disease-before-birth

this article talks about altering the gene that causes a lung-disease using CRISPR technology. the amnionic fluid of mice fetus carrying a mutated SFTPC gene, helps prevent the lung from collapsing when exhaling, were injected with CRISPR-Cas9. The injection only targeted the lungs and didn't affect any other organs. Over 22 percent of the mice survived from the disease. Also the infection didn't affect the reproductive cells of the fetus which means it can not be inherited by future generations. More research and experiments are being held to further improve the technology and increase the rate of success. Scientists are also trying to repair the gene rather than inactive it so they can apply this operation with different delivery methods on humans as humans need a functional SFTPC gene.



I found the articles really interested as the scientist could actually target specific organs such as the lungs and even cells within the lungs. I also admire their persistence on not only increasing the rate of success but also making sure that there is no negative affects on the mother of the embryo. It is a huge advancement in science and technology to be able to treat a disorder in the womb to save a fetus. I hope that the scientists can find a safe, efficient, ethical method to do the same treatment for humans soon.

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.