Gene Therapy

April 6, 2021 · Leave a Comment

Mayo researchers reveal gene therapy path for treating children with rare, fatal genetic disease

By Susan Murphy Susan Murphy (@susanmurphy)

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 […]

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Tags: #gene therapy, rare disease


January 28, 2021

First hybrid gene therapy shows early promise in treating long QT syndrome

By Colette Gallagher Colette Gallagher (@colettegallagher)

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 […]

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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 Susan Murphy (@susanmurphy)

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, […]

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Tags: artificial intelligence, Cancer Research, Genetics, genomics, individualized medicine, multi-omics, Research


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