Advancing the Science

Mayo Clinic Medical Science Blog – an eclectic collection of research- and research education-related stories: feature stories, mini news bites, learning opportunities, profiles and more from Mayo Clinic.

Items Tagged ‘gene therapy’

April 22, 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: Center for Individualized Medicine, gene therapy, genomics, precision medicine, rare disease


February 16, 2021 · Leave a Comment

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 1 long […]

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Tags: cardiology, cardiovascular disease, cardiovascular medicine, Center for Individualized Medicine, Center for Regenerative Medicine, gene therapy, long QT syndrome, Michael Ackerman, molecular pharmacology and experimental therapeutics, pediatric cardiology, pediatric research, sudden cardiac death


December 8, 2020 · Leave a Comment

How COVID-19 empowered regenerative sciences students for success

By Susan Buckles Susan Buckles (@susanbuckles)

Training the workforce of the future is a strategic priority of Mayo Clinic’s Center for Regenerative Medicine. Sometimes the most valuable lessons come from outside the classroom. This year, regenerative sciences students readily adapted to a stealthy foe that exploded on the scene, seemingly from nowhere. The COVID-19 pandemic triggered a rapid conversion to online […]

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Tags: CAR-T, Center for Regenerative Medicine, Claudia Manriquez Roman, COVID-19, Dileep Monie, gene therapy, immunology, Mayo Clinic Alix School of Medicine, Mayo Clinic Graduate School of Biomedical Sciences, medical research, Naga Rallabandi, research education


February 7, 2019 · Leave a Comment

Eva Galanis, M.D.-Bitten by the virotherapy bug

By Advancing the Science contributor Advancing the Science contributor (@advancingthescience)

Every day more than 1,600 Americans die from cancer. Most of them have cancer that can’t be cured with traditional methods — surgery, radiation and chemotherapy. The father of Evanthia Galanis, M.D., was one of them. He died in the late 1990s from melanoma when his daughter was a junior faculty member at Mayo Clinic. “My father would have […]

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Tags: basic science, cancer, clinical trials, Eva Galanis, gene therapy, hematology, immunotherapy, Mayo Clinic Cancer Center, measles virus, oncology, SPORE, virotherapy


January 10, 2019 · Leave a Comment

Gene therapy: potential and pitfalls

By Susan Buckles Susan Buckles (@susanbuckles)

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 […]

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Tags: Center for Individualized Medicine, David Deyle, gene mutation, gene therapy, Saad Kenderian


October 18, 2018 · Leave a Comment

What is the impact on health care of genome editing?

By Colette Gallagher Colette Gallagher (@colettegallagher)

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 […]

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Tags: Center for Individualized Medicine, CRISPR, gene therapy, genome, Individualizing Medicine Conference, Megan Allyse


October 9, 2018 · Leave a Comment

Diabetes 3, 2, 1

By Jennifer Schutz Jennifer Schutz (@jenniferschutz)

Often people with diabetes have brain changes that are hallmarks of both Alzheimer’s disease and vascular dementia. Some researchers believe that each condition fuels the damage caused by the other. That link may occur as a result of the ways that type 2 diabetes affects the ability of the brain and other body tissues to […]

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Tags: Alzheimer's disease, Center for Regenerative Medicine, diabetes, endocrinology, gene therapy, Guojun Bu, stem cells


August 23, 2018 · Leave a Comment

Using the body to recognize and attack cancer

By Susan Buckles Susan Buckles (@susanbuckles)

This article originally appeared in the Center for Individualized Medicine blog on July 23, 2018 For as long as he can remember, Saad Kenderian, M.B., Ch.B., wanted to be a physician. Nothing could blunt his resolve –not even when improvised explosive devices, bombs and trappings of war put the medical school in his native Baghdad, […]

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Tags: cancer, CAR-T, Center for Individualized Medicine, gene therapy, immunotherapy, Mayo Clinic Cancer Center, Saad Kenderian


November 7, 2017 · Leave a Comment

No moss here – Mayo Clinic research websites rolling along

By Elizabeth Zimmermann Elizabeth Zimmermann (@elizabethzimmermann)

They say that a rolling stone gathers no moss, and at Mayo Clinic, our research and research education programs are always on the move. It’s hard to keep track of all the exciting work being done to help improve health for our patients, or the way they – and patients everywhere – experience health care. […]

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Tags: basic science, Christopher Evans, Dev Mukhopadhyay, DNA, Eva Galanis, gene therapy, Jim Maher, Michael Krowka, nanomedicine, osteoarthritis, transplant


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