Scientists Use LowKey CRISPR Technique to End Marfan Syndrome

first_img A new gene-editing method could prevent humans from passing on Marfan Syndrome, a genetic disorder that affects the body’s connective tissue.Researchers at China’s Guangzhou Medical University and ShanghaiTech University developed a CRISPR technique intended to reduce the odds (currently 50/50) of transmitting the ailment from parent to child.About one in 5,000 people have Marfan Syndrome, which manifests as tall, thin limbs, flexible joints, and scoliosis. The most serious complications involve increased risk of mitral valve prolapse and aortic aneurysm.In its current form, CRISPR/Cas9 is poised to become the gene-editing tool of the future.It works a lot like a word processor’s cut-and-paste function: Scientists target a specific spot in an organism’s genetic code, cut the DNA strands, and insert a new gene or let the strands self-repair.But the technology may not be all its cracked up to be.In an effort to avoid developmental snags, the Chinese researchers tried a different approach—”base editing.” The two-year-old procedure allows users to swap DNA letters, and, fingers crossed, cause fewer unwanted edits.In the case of Marfan Syndrome, the fix seems easy: Just change the flawed G in the FBN1 gene to a healthy A.According to the study, published in the journal Molecular Therapy, all went well; researchers were able to correct the mutation in 18 viable human embryos.Two early-stage organisms, however, exhibited unintended alterations—a C changed to a T.Still, the team remains cautiously optimistic.“Overall, this pilot study provided proof of concept, and opened the potential of base editing-based gene therapy,” Xingxu Huang of ShanghaiTech University told STAT, as reported by Futurism. “Nevertheless, there is still a long way to go to use it in IVF clinics.”Last year, researchers in Oregon became first in the US to genetically modify human embryos. And while scientists adopt CRISPR to fight diseases like HIV and autism, the gene-editing tool remains controversial. Find out more about it here.Let us know what you like about Geek by taking our survey. CRISPR-Modified Babies Cursed With Short LifespanAntidote to Deadly Box Jellyfish Venom Discovered Stay on targetlast_img read more