Disease Focus: Skeletal/Smooth Muscle disorders
Clinical Translation of hESC-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating DM1
Translational Candidate We engineered a human embryonic stem cell-secreted signaling protein into a biologic for treatment of skeletal muscle disorders. Area of Impact Skeletal muscle disorders (including DM1 and sarcopenia) remain major unmet needs that require treatments restoring muscle strength and function. Mechanism of Action Our animal data demonstrate an endocrine stimulation by our biologic […]
Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence
Translational Candidate Smooth muscle cell progenitors (pSMCs) differentiated from patient iPSCs which is injected into the urethral muscle to regenerate a a weak urethra. Area of Impact Surgery for urinary incontinence is effective in 80% of patients. Our target is those who failed surgery (20%), or those who cannot undergo surgery. Mechanism of Action Our […]
2024 Center for Dental, Oral, & Craniofacial Tissue & Organ Regeneration (C-DOCTOR) Annual Spring Conference
2023 Center for Dental, Oral, & Craniofacial Tissue & Organ Regeneration (C-DOCTOR) Annual Spring Conference
Phase I Treatment of Urethral Strictures in Humans
Therapeutic Candidate or Device An engineered urethral segment comprised of autologous urothelial, smooth muscle cells, and progenitor cells within a tubular scaffold. Indication Autologous engineered urethral constructs are for the replacement of urethral strictures that are too long for treatment by conventional methods. Therapeutic Mechanism The autologous engineered urethras are intended to replace the entire […]
A Double-Blind Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Progenitor Cells for the Treatment of Dysphagia
Therapeutic Candidate or Device Autologous Muscle Derived Progenitor Cells (AMDC) isolated from skeletal muscle biopsy Indication Subjects with dysphagia (swallowing difficulties) that develops following treatment for head and neck cancer Therapeutic Mechanism Autologous Muscle Derived Progenitor Cells differentiate to form new muscle fibers, engraft into existing myofibers, and have been shown to increase muscle diameter […]
Technologies to improve in vivo function of transplanted stem cells
Stem cell-based therapy is recognized as a promising therapeutic approach for treating various diseases that are currently intractable. One strategy in regenerative medicine is to transplant stem cells or their differentiated derivatives to regenerate the damaged tissues or halt tissue degeneration. Human embryonic stem cells and human induced pluripotent stem cells having the potential to […]
Molecular regulation of stem cell potency
The field of stem cell biology as it applies to regenerative medicine requires a detailed understanding of what controls stem cell function. Our fundamental interest is in the molecular pathways that control how potent as a stem is in terms of its ability to make new tissues in response to injury and disease. We have […]
Combination therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) affects 1 in every 3,500 boys worldwide. DMD is caused by mutations in the gene encoding dystrophin, a protein key to muscle health. DMD patients are typically weaker than normal by age 3, and with progressive muscle weakness most loose the ability to walk by age 11. DMD progresses to complete […]
Engineered iPSC for therapy of limb girdle muscular dystrophy type 2B
Limb girdle muscular dystrophy type 2B (LGMD 2B) is a form of muscular dystrophy that leads to muscle degeneration and disability. In LGMD 2B, a vital muscle protein is mutated, and its absence leads to progressive degeneration of muscles in the body that are needed for mobility. To create a therapy, we will provide a […]