Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy

Children and adolescents come from around the world to be treated at M Health Fairview Masonic Children's Hospital.  Our Blood and Marrow Transplantation & Cellular Therapy (BMT&CT) Program at the University of Minnesota is the oldest and one of the largest and most respected pediatric blood and marrow transplant research programs in the world. We performed the first successful BMT in 1968 and we are still delivering firsts in BMT&CT therapy, patient care, and research. 

The M Health Fairview Masonic Children's Hospital is an international leader in the treatment of epidermolysis bullosa (EB), an often life-threatening skin-blistering disorder.

Our Hemoglobinopathies Program stands at the forefront of treatment and research for patients with sickle cell disease (SCD) and thalassemia. We are dedicated to providing state-of-the-art curative therapies, including advanced hematopoietic stem cell transplantation and innovative gene therapy.

The ALD and Leukodystrophy Center at the University of Minnesota has unsurpassed experience treating leukodystrophy patients. Our physicians lead the field in several areas of research and continue to explore innovative ways to improve treatment success.

Our MPS Center is internationally recognized for our expertise, innovative treatment approaches, and research. Patients are cared for by experts in the areas of audiology, blood and marrow transplantation, cardiology, ear, nose and throat, endocrinology, genetics, neurology, neuropsychology, neuroradiology, ophthalmology, orthopeadic surgery, and pulmonology. This multidisciplinary team approach ensures that the care we provide best meets the unique needs of each patient and family.

Immunobiology and transplantation are the main research interest in the Blazar Laboratory. Project areas include: Graft-versus-host disease, regulatory T cells, immune recovery and regeration post-transplant, graft-versus-leukemia and genetic manipulations and reprograming of immature and mature cells.

The Bosnakovski lab is focused on tissue regeneration and understanding the molecular and cellular mechanisms of FSHD and sarcomas. The current work includes exploring the molecular and cellular effects of DUX4 expression in skeletal muscle and studying antifibrotic drugs on an FSHD mouse model. The lab is also modeling the initiation of CIC-DUX4 pediatric sarcomas and exploring novel epigenetic-targeted therapies for Ewing sarcomas.

Congenital heart defects and muscular dystrophies are common hereditary diseases affecting children, but effective treatments for most of these disorders are scarce. In the Chan Laboratory, they are working on deciphering the molecular regulation of how these diseases develop and establishing stem cell-based therapies to combat them.

The Laboratory of Experimental Tissue Regeneration, led by Dr. Michael Kyba, focuses on factors regulating tissue degeneration and regeneration, with a view towards using cell therapy for regenerative therapy.

The Panoskaltsis-Mortari laboratory is centered on the field of regenerative medicine. We study tissue engineering from stem cells using two approaches:

1. Decellularized whole organ scaffolds integrated with sophisticated bioreactors
2. 3D Bioprinting using extrusion, suspension, and laser-assisted techniques

The Tolar Laboratory focuses on finding new ways of treating children with lethal diseases. Additional research areas include:

• Reducing the negative effects of stem cell transplantation
• Creation and use of induced pluripotent stem cells
• Gene therapy using gene addition 
• Gene editing