{"id":79839,"date":"2025-10-14T12:11:06","date_gmt":"2025-10-14T12:11:06","guid":{"rendered":"https:\/\/www.newsbeep.com\/ie\/79839\/"},"modified":"2025-10-14T12:11:06","modified_gmt":"2025-10-14T12:11:06","slug":"mizzou-researcher-creating-a-new-way-to-treat-genetic-disorders","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/ie\/79839\/","title":{"rendered":"Mizzou researcher creating a new way to treat genetic disorders"},"content":{"rendered":"

By Brian Consiglio<\/a><\/p>\n

\"Dunpeng<\/a>Dunpeng Cai <\/p>\n

October 14, 2025
Contact: Brian Consiglio, consigliob@missouri.edu<\/a>
Photo by Abbie Lankitus<\/p>\n

The University of Missouri is breaking new ground in the treatment of genetic disorders, and it\u2019s starting with Marfan syndrome \u2014 a potentially fatal genetic disorder that weakens the aorta, the heart\u2019s main artery.<\/p>\n

With a grant from the American Heart Association, Mizzou\u2019s Dunpeng Cai is creating a novel RNA-based therapy to treat Marfan syndrome, which is caused by a mutation in the fibrillin-1 protein. Left untreated, it can lead to aortic aneurysms \u2014 tears or ruptures in the aorta \u2014 which can be deadly.<\/p>\n

One current treatment, DNA editing, is risky because it can cause irreversible damage if a mistake is made. Cai\u2019s approach is safer by targeting RNA instead.<\/p>\n

\u201cThe goal of our new therapy is to correct the mutated RNA for those with Marfan syndrome, thus producing the fibrillin-1 protein in its correct form, ultimately curing this genetic disorder,\u201d Cai, an assistant professor in the School of Medicine<\/a>, said.<\/p>\n

With the resources of the Roy Blunt NextGen Precision Health<\/a> building at Mizzou, Cai is well-positioned to eventually take this research toward clinical trials. If successful, this RNA editing method could be applied to treat a wide range of genetic disorders, including Huntington\u2019s disease, Down syndrome and sickle cell disease.<\/p>\n

The RNA-based therapy may also have implications for cancer treatment. Some patients do not respond to current therapies due to specific genetic mutations. Cai\u2019s system could one day correct those mutations, making treatments more effective.<\/p>\n

Cai\u2019s work is part of Mizzou\u2019s broader push into precision medicine \u2014 tailoring treatments to the unique genetic makeup of individual patients. By sequencing a patient\u2019s genes, Cai can pinpoint the exact location of the mutation and customize the RNA-based therapy accordingly.<\/p>\n

\u201cThis innovative RNA-based approach could be a game-changer for precision medicine,\u201d Cai said. \u201cMizzou is the perfect place for this research, as the Genomics Technology Core helps make this research successful.\u201d<\/p>\n

Cai\u2019s interest in precision medicine began during his doctoral studies in pharmacology, when he questioned why traditional drugs worked well for some patients with genetic disorders but not for others. He discovered that while patients may share the same affected protein, the precise mutations can differ \u2014 a realization that inspired his current RNA-focused work.<\/p>\n

Cai completed both his doctoral studies and postdoctoral training at Mizzou before becoming a faculty member. He credits Shiyou Chen and Stephen Barnes in the School of Medicine for their mentorship and encouragement.<\/p>\n

\u201cSearching for new ways to improve the quality of life for those with genetic disorders is what motivates my work,\u201d Cai said. \u201cSince those with Marfan syndrome have weaker connective tissue in their heart, bones, lungs and eyes, they get tired more easily and may not be able to exercise much or do physical activities they enjoy. Helping people is my current goal, and if this new approach works, Marfan syndrome is just the tip of the iceberg.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"By Brian Consiglio Dunpeng Cai October 14, 2025Contact: Brian Consiglio, consigliob@missouri.eduPhoto by Abbie Lankitus The University of Missouri…\n","protected":false},"author":2,"featured_media":79840,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[25],"tags":[254,61,60,82],"class_list":["post-79839","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","tag-genetics","tag-ie","tag-ireland","tag-science"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts\/79839","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/comments?post=79839"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/posts\/79839\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/media\/79840"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/media?parent=79839"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/categories?post=79839"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/ie\/wp-json\/wp\/v2\/tags?post=79839"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}