At the University of Minnesota, our GT-COE is a leader in pioneering gene therapy clinical trials, offering hope and cutting-edge treatments for patients with rare and complex conditions. We focus on advancing therapies for conditions such as adrenoleukodystrophy (ALD), sickle cell disease, mucopolysaccharidosis type I, and metachromatic leukodystrophy.
Diseases
Adrenoleukodystrophy (ALD)
Adrenoleukodystrophy is X-linked in inheritance, and therefore affects almost exclusively boys at a rate of 1 in 20,000. There are various forms of ALD, including adrenal insufficiency (affecting the adrenal glands), cerebral ALD (affecting the brain), and AMN (adrenomyeloneuropathy; affecting the spinal cord). The University of Minnesota is one of the most experienced centers in the world in the assessment and treatment of patients with ALD.
The University of Minnesota was one of only three sites to offer the gene therapy treatment, Skysona, as a clinical trial and we are proud to be a Qualified Treatment Center to treat ALD now that it is FDA approved. As one of the highest enrolling site of the clinical trial, we have the unique experience and expertise to effectively manage treatment.
The ALD National Registry at the University of Minnesota is the world’s largest dataset of clinical information, MRI scans, and biological samples related to ALD. We believe this will help researchers throughout the world better understand the natural history of the disease, including factors related to the onset and progression of disease.
For information about our Comprehensive Adrenoleukodystrophy Clinic please visit our Leukodystrophy Center webpage.
B-Thalassemia
Beta-thalassemia is an inherited blood disorder that impacts the ability for the body to effectively produce hemoglobin, which can lead to anemia. Thalassemia is more common in individuals with ancestry from the Mediterranean, Southeast Asia, and Africa. The condition is passed from parents to children through mutations in the genes responsible for hemoglobin production.
In 2025, the University of Minnesota will be activated as a Qualified Treatment Center for the gene therapy, Zynteglo.
Metachromatic Leukodystrophy (MLD)
Metachromatic Leukodystrophy is a rare genetic disorder that affects coordination, strength and cognition. There are three types of MLD, late infantile (evident between 6-30 months of age), juvenile (evident between 30 months and 17 years old), and adult (evident between 17-40 years old).
The University of Minnesota performed the first gene therapy procedure for MLD in the United States in 2021. With the successful treatment of the first patient, we subsequently treated 5 more pediatric patients, contributing to the FDA approval of Lenmeldy. As of December 19, 2024, the University of Minnesota is one of only two Qualified Treatment Centers in the U.S. to use Lenmeldy for the treatment of MLD.
Sickle Cell Disease
Sickle Cell Disease is an inherited blood disorder that affects the shape and flexibility of the red blood cells. Sickle Cell can cause anemia, pain crises, swelling in the hands and feet, frequent infections, delayed growth, and vision problems. Serious complications can include stroke, acute chest syndrome, organ damage, and more. The NIH reports over 100,000 people are affected in the United States.
Our team of experts are dedicated to advancing new treatment options for Sickle Cell Disease by collaborating with providers, researchers and community organizations. Our research has contributed to groundbreaking gene therapies and we are excited to be a treatment site in the near future!