Disease Focus: Skin Disease
Scaffold for dermal regeneration containing pre-conditioned mesenchymal stem cells to heal chronic diabetic wounds
The goal of our CIRM-funded Early Translational (ETA) grant was to engineer a product to improve healing in diabetic foot ulcers, a devastating consequence of diabetes that occurs in about 25% of all diabetic patients and is responsible for most leg or foot amputations. More than 6 million people in the US and up to […]
A Chromatin Context Tool for Predicting iPS Lineage Predisposition and Tissue Graftability
Induced pluripotent stem (iPS) cells are cells derived from skin that closely resemble embryonic stem (ES) cells and can be coaxed into many different types of cells such as nerve cells, heart cells, liver cells, and also back to skin cells. One major bottleneck in the field is our ability to coax the cells into […]
Tunable hydrogels for therapeutic delivery of multipotent stem cells.
Rationale: Skin ulcers represent the largest economic burden of all skin diseases. Human adult stem cell therapies for the treatment of chronic wounds have shown considerable promise. However, a delivery system is needed before they can be used clinically. Hydrogels have emerged as a promising vehicle for stem cell delivery because their 3-D structure and […]
Regulation of Epidermal Self-Renewal and Differentiation by Histone and DNA Demethylases.
Tissue specific stem and progenitor cells exist to replenish the tissue it resides during normal homeostasis or during regeneration from a wound. Disease and aging leads to a depletion of these stem and progenitor cells, which can impede the ability of the body to regenerate itself. Thus, an understanding of the mechanisms of how tissue […]
Bone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds
Diabetic foot ulcers (DFU), chronic, non-healing wounds on the feet of diabetic patients, present a serious challenge to global health. These ulcers affect between 15-25% of the 18-21 million Americans who have diabetes (world-wide incidence of diabetes: 366 million people). DFUs have a huge impact on our health care system, not only in terms of […]
iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa
Genetic skin diseases constitute a diverse group of several hundred diseases that affect up to 2% of the population and include common disease such as psoriasis, atopic dermatitis, and wound healing. Patients with one genetic disease, dystrophic Epidermolysis bullosa (EB), lack a normal collagen VII (COL7A1) gene and suffer from debilitating blistering and scarring that […]
Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis
Research Objective Locally delivered formulation of RSpo1 protein as an activator of Lgr5+ epithelial stem cells in chemotherapy- or radiation therapy-induced oral mucositis Impact Oral mucositis Major Proposed Activities RSpo1 formulation design and selection for optimal oral delivery Activation of Wnt pathway by formulated RSpo1 in-vitro Production of RSpo1 protein Oral stem cell expansion by […]
A universally applicable skin sheet for Dystrophic Epidermolysis Bullosa via next-generation gene editing, iPS cell technology and tissue engineering
Research Objective We will develop a cell therapy for a rare skin disease. Patient-derived iPS cells will be genetically corrected and differentiated into epithelial sheets to be grafted on skin wounds. Impact In this proposal we will develop a universal genetic correction strategy for all COL7A1 which will be a prerequisite for the commercial viability […]
Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis
Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue. Unmet Medical Need Systemic sclerosis is an incurable disease with no […]
DEBCT: Genetically Corrected, Induced Pluripotent Cell-Derived Epithelial Sheets for Definitive Treatment of Dystrophic Epidermolysis Bullosa
Translational Candidate DEBCT is an autologous iPS-derived COL7A1-corrected keratinocyte graft indicated for the treatment of all chronic open wounds in patients with RDEB. Area of Impact RDEB patients lack type VII collagen and have chronic wounds that lack an abundance of keratinocyte stem cells. DEBCT skin grafts will close wounds. Mechanism of Action RDEB patient […]