ISCRM Associate Director Nate Sniadecki, PhD, Professor of Mechanical Engineering and Hichem Tasfaout, PhD, Acting Assistant Professor, Neurology have received prestigious awards from the John H. Tietze Foundation Trust that will help fuel promising research underway in their labs.
Engineering 3D Tendons
Tendon pathologies are a common clinical problem involving significant pain and disability. For example, the incident rate for rotator cuff tears is one in five people and requires over 200,000 surgeries annually. With the increase in life expectancy, the instances of tendon injuries will continue to rise, placing a financial strain on healthcare systems.
Much of the research on tendon healing has relied on studies with rodents, but there is a biological difference between rodent cells and human cells. With funding from the 2024 John H. Tietze Stem Cell Scientist Award, Dr. Nate Sniadecki and his lab will develop an in vitro, mechanobiological model of human tendons. These engineered tendon models have the potential to be used to test new therapies, model genetic diseases, or build replacement tissue materials.
The award will enable the researchers to, we will examine the maturation of stem cell-derived tenocytes in a mechanobiological 3D environment and recapitulate the bone-tendon interface (enthesis) using a suspended open microfluidics-based tissue engineering approach. The lab will explore whether tendon cells cultured in 3D mature faster than they do in 2D environments and examine how closely these engineered tendons resemble human tissues.
Importantly, development of a sophisticated ETC platform will enable future studies in the Sniadecki lab on tendon injury mechanisms and surgical engraftment studies for tissue replacement therapy.
Testing a New Way to Treat Muscle Disorders
Gene replacement using Adeno-associated viral (AAV) vectors represents a promising therapeutic approach for many genetic diseases. Recent advances in this field have led the FDA (Food and Drug Administration) and EMA (European Medicines Agency) to approve several gene therapies for life-threatening genetic disorders such as spinal muscular atrophy.
The Jaconette L. Tietze Young Scientist Award will enable Dr. Hichem Tasfaout to study how AAV vectors could best be used to treat Ryanodine Receptor 1-related myopathies, a family of disorder that present muscle weakness, motor delay resulting in difficulties walking and climbing stairs, scoliosis, muscle pain (myalgia), muscle cramps, and fatigue.
RYR1 disorders are caused by genetic mutations in the RYR1 gene, which abolish the expression of RYR1. Functional copies of the faulty RYR1 gene can, in theory, be delivered to the affected muscles using AAV vectors, if the healthy gene could be packaged into the relatively small vector.
Funding from the Tietze Award will allow Dr. Tasfaout to investigate a new gene delivery method that take advantage of a system, developed in the Chamberlain Lab, known as SIMPLI-GT. This technology enables the expression of large proteins by directing several protein fragments to affected muscle cells and then fusing them into one functional protein using small molecules named split intein. This novel gene therapy approach aims to express the missing RYR1 protein and, if successful, would benefit all patients with RYR1-myopathies.
About the Tietze Research Awards
John H. Tietze Stem Cell Scientist Award
The John H. Tietze Stem Cell Scientist Award is a one-year award of $50,000 to support the research of any UW FACULTY member of ISCRM who is pursuing novel preliminary experiments, where the grant might provide sufficient stimulus to enable the research to advance to the point of being competitive for external funding. The research should involve or be relevant to some aspect of stem or progenitor cell biology or therapies.
Jaconette L. Tietze Young Scientist Award
The Jaconette L. Tietze Young Scientist Award is also for one year of support of $25,000. Preference will be given a junior faculty (including Acting Instructor level) located at the UW who has not yet received major external funding (such as an R01). The research should involve or be relevant to some aspect of stem or progenitor cell biology or therapies.