Disease Focus: Other
Embryonic stem cell-based generation of rat models for assessing human cellular therapies
Heart failure, diabetes and neurodegenerative diseases are among the leading causes of death and disability worldwide. These diseases are characterized by the loss of specific cell types and can be treated and potentially cured with stem cell-based therapies. Before human stem cells can be used in clinical trials, however, their safety and efficacy need to […]
A small molecule tool for reducing the malignant potential in reprogramming human iPSCs and ESCs
This research project aims to solve a key bottleneck in the use of differentiated human embryonic stem cells and induced pluripotent stem cells for the regeneration and replacement of diseased or damaged tissues. This bottleneck is the potential of unintended transplants containing failed-to-differentiate stem cells developing into benign growths called teratomas, or worse, malignant teratocarcinomas. […]
Biophysical Determinants of Early Embryonic Stem Cell Fate Specification
Regenerative therapies require effective differentiation of stem cells to cell types that are functionally identical to those found in vivo. Many current differentiation protocols merely involve optimization of proteins added to the culture media, but do not consider the microenvironmental context in which cells differentiate during development or tissue repair. When we include the biophysical […]
Single Molecule Biophysics and Biology of Cellular Identity
One of your earliest childhood biology lessons probably occurred when your body demonstrated to you that your skin is an organ that is able to self-regenerate. Indeed wound healing is a fascinating process in which cells carry out a precise and complex choreography that includes cellular differentiation and regulation of gene expression. Our lab studies […]
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
hPSC-derived enteric ganglioids for cell therapy in gastrointestinal motility disorders
Research Objective The proposed aims will enable the generation, purification and characterization of enteric neurons from diverse hiPSCs and assessment of their efficacy for cell therapy in GI motility disorders. Impact This proposal addresses a significant unmet clinical need for a cell therapy approach for gastrointestinal motility disorders such as Hirschsprung disease, achalasia and gastroparesis. […]
Novel antisense therapy to treat genetic forms of neurodevelopmental disease.
Research Objective We propose to discovery and evaluate antisense gene therapy for specific mutations underlying debilitating or life-threatening neurodevelopmental diseases including epilepsy and autism syndromes. Impact The conditions are four specific neurodevelopmental syndromes where mutations are well suited to ASO therapy. The bottlenecks are current lack of cellular evidence for ASOs to impact disease course. […]
AAV-dCas9 Epigenetic Editing for CDKL5 Deficiency Disorder
Research Objective We propose a gene therapy for the treatment of a severe infantile epilepsy called CDKL5 Deficiency Disorder using CRISPR-mediated epigenetic editing Impact A transformative treatment for females affected by CDKL5 Deficiency Disorder in addition a platform for the approximately 38 other X-linked intellectual disabilities that predominately affect females Major Proposed Activities Validation of […]
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 […]
Non-Toxic, Highly-Effective Bioinspired Cryoprotectants for On-Demand Stem Cell Therapies
Research Objective An advanced technology is sought to replace the toxic, ineffective and highly processed components in legacy cryopreservation media. This technology is a fundamentally novel non-toxic freezing media. Impact The transport and storage of stem cell therapies is crippled by freezing media with poor cell preservation. Significantly improved freezing media would directly increase therapy […]