Items Tagged ‘#gene therapy’
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
November 19, 2018
Gene therapy: potential and pitfalls
Research is advancing gene therapy as a possible treatment or eventual cure for genetic diseases that bedevil modern science. Gene therapy was conceived over 20 years ago, and until recently, remained largely in the research lab. But gene therapy products are now beginning to be approved by the U.S. Food and Drug Administration for clinical […]
Tags: #Brittle bone disease, #CAR T-cell therapy, #Dr. David Deyle, #gene therapy, Dr. Saad Kenderian, Mayo Clinic Center for Individualized Medicine, medical research
September 24, 2018
CIM CON — What is the impact on health care of genome editing?
Although Mayo Clinic does not use genome editing as part of any treatment in the medical practice, genome editing has promise for treating and even curing previously intractable disorders, such as Duchenne muscular dystrophy. Genome editing, via methods like CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and CRISPR-associated protein) can be used to facilitate the […]
Tags: #CIMCon18, #CRISPR-Cas9, #Dr. Megan Allyse, #Dr. Shondra Pruett-Miller, #Dr. Stephen Tsang, #duchenne muscular dystrophy, #gene therapy, center for individualized medicine, gene editing, genomics, mayo clinic