Disease Focus: Skeletal/Smooth Muscle disorders


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 […]

Characterization of Human Skeletal Muscle Stem Cells for Clinical Application

Skeletal muscle makes up 40% of our bodies, dictates our form, is responsible for our ability to move, express ourselves, eat, breath and to look around. Restoration or preservation of the body’s normal form and function is the central goal of regenerative medicine and the central focus of my clinical specialty of plastic and reconstructive […]

Combination Therapy to Enhance Antisense Mediated Exon Skipping for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy and one of the most common fatal genetic disorders. Approximately one in every 3,500 boys worldwide is affected with DMD. Extrapolating from population based studies, there are over 15,000 people currently living with DMD in the US. DMD is a devastating and incurable muscle-wasting disease […]

Local Delivery of Rejuvenated Old Muscle Stem Cells to Increase Strength in Aged Patients

As humans age, the ability to regenerate skeletal muscle tissue is impaired. Injuries to the musculoskeletal system that require extended periods of immobilization lead to muscle atrophy and are particularly devastating to the elderly population. Loss of skeletal muscle mass and function reduces mobility, which negatively affects quality of life, and increases the risk of […]