Gene Therapy
February 27, 2023
Mayo Clinic discovery leads to life-changing treatment for young girl with ultra-rare disease
By Susan Murphy 
Months after young Maggie Carmichael started taking an experimental drug for her ultra-rare genetic disease, she was able to trade in her wheelchair for a walker. The 9-year-old Mayo Clinic patient even took a few of her first-ever steps on her own. She also became better at feeding herself, her speech improved and she started coloring in the lines.
Tags: #Dr. Eva Morava-Kozicz, center for individualized medicine, DNA Testing, genomic medicine, personalized medicine
October 18, 2022
In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS). LQTS is a genetic heart disease that can potentially cause fast, chaotic heartbeats. These rapid heartbeats might trigger people to […]
Tags: center for individualized medicine, gene therapy, genomic medicine, individualized medicine, mayo clinic, personalized medicine
December 28, 2021
10 Significant Mayo Clinic Center for Individualized Medicine studies of 2021
By Susan Murphy
It has been an impactful year of genomic and multi-omic research and scientific discoveries in Mayo Clinic’s Center for Individualized Medicine. While much focus in 2021 was centered on advancing the knowledge of COVID-19, Mayo scientists and physician have also worked to develop individualized treatments, prevention measures and diagnostics for patients with rare and undiagnosed diseases, cancer and other illnesses.
Tags: center for individualized medicine, DNA Testing, gene sequencing, Genetics, genomic medicine, genomics
December 7, 2021
Pushing the Limits of Gene Therapy
By Susan Murphy
Dr. Ackerman is part of a cadre of researchers at Mayo Clinic who are pushing the limits of what is possible with gene therapy. They are looking beyond simply saving lives to improving the quality of life for people dealing with heart disease, liver disease and arthritis.
April 6, 2021
Mayo researchers reveal gene therapy path for treating children with rare, fatal genetic disease
By Susan Murphy
A gene therapy strategy developed by Mayo Clinic researchers could offer a potential treatment for a rare and fatal genetic disease that often sickens babies in their first days of life. The disease, propionic acidemia, occurs in 1 in 100,000 live births in the U.S. There is no cure. “As soon as the babies start […]
Tags: #gene therapy, rare disease
January 28, 2021
First hybrid gene therapy shows early promise in treating long QT syndrome
In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition. Researchers demonstrated its potential therapeutic efficacy in two in vitro model systems using beating heart cells reengineered from the blood samples of patients with type […]
Tags: #Dr. Michael Ackerman, #gene therapy, #News release, Circulation, congenital heart disease, Genetics, long QT syndrome, mayo clinic, Mayo Clinic Center for Individualized Medicine, Minnesota News Releases
December 24, 2020
2020 Mayo Clinic Precision Medicine Advances
By Susan Murphy
This year, Mayo Clinic’s Center for Individualized Medicine was at the forefront of COVID-19 research, working to unravel the complexities of the virus in order to discover life-saving treatments and prevention — from testing to building a pandemic response biobank to identifying COVID-19 strains within individual patients. But even in the midst of the pandemic, […]
Tags: #Artificial Intelligence, Cancer Research, Genetics, genomics, individualized medicine, multi-omics, Research