Disease Focus: Respiratory Disorders
Pro-healing biomaterial for treating lung inflammation associated with COVID-19
Research Objective Pro-healing biomaterial to treat lung inflammation and promote recovery. Impact COVID-19 associated acute respiratory distress syndrome Major Proposed Activities Evaluate biomaterial for ability to reduce lung inflammation in rodent model Evaluate biomaterial for ability to recruit stem cells in the lung Evaluate biomaterial for ability to improve lung function in rodent model Evaluate […]
Stem cell-based rapid identification of SARS-CoV-2 T cell epitopes and T cell receptors for therapeutic use
Research Objective We will identify SAR-CoV-2 T cell epitopes for vaccine development and specific TCRs for adoptive T cell therapy using a stem cell-based platform to generate specialized dendritic cells in vitro. Impact New methods to rapidly identify T cell epitopes would greatly accelerate development of vaccines and TCR-based therapeutics, and in the setting of […]
Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]
Stem Cells for Lung Diseases – Overcoming Barriers to Find New Therapies
Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells.
Research Objective Novel selective pharmacological strategy targeting senescent lung stem cells Impact Idiopathic pulmonary fibrosis along with other interstitial and age-related lung diseases Major Proposed Activities Chracterize senescence cells, including senescent stem and progenitor cells in the lung of patients affected by idiopathic pulmonary fibrosis Screening of a Rubedo Life Science library of senolytic small […]
Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis
Research Objective We propose to develop mesenchymal stem cell derived extracellular vesicles (MSC-EV) as treatment for lung fibrosis Impact MSC-EV are promising for several lung diseases, but we need to better understand how they work, where they go in the body, and whether there is a subset of MSC-EV with better efficacy Major Proposed Activities […]
Genome Editing to Correct Cystic Fibrosis Mutations in Airway Stem Cells
Research Objective Gene corrected autologous airway epithelial stem cells from patients with cystic fibrosis to be used as cell and gene based therapy for chronic sinus disease Impact The proposed studies would provide an innovative, readily applied primary stem cell based approach with gene correction to treat chronic sinusitis in CF, a debilitating airway disease. […]
Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration
Research Objective We will generate human airway stem cells by direct transcriptional reprogramming of fibroblasts. We will use these induced airway stem cells to model motile cilia disease in a dish. Impact Generating airway stem cells through reprogramming will create a scalable and editable cell line from which we can derive airway epithelium, thus enabling […]
Telomerase mRNA for short telomere related pulmonary fibrosis
Translational Candidate Nucleoside-modified mRNA encoding telomerase reverse transcriptase (TERT) encapsulated in a lung-targeting lipid nanoparticle delivered intravenously. Area of Impact 1) Idiopathic pulmonary fibrosis (IPF) and 2) Other diseases and conditions caused or exacerbated by short telomeres. Mechanism of Action TERT mRNA LNPs transiently extend the telomeres of the alveolar epithelial cells of the lung, […]
Tissue Engineered Recellularized Laryngotracheal Implants
The goal is to bring a safe and effective therapy to adult patients with critical narrowing of the upper windpipe (trachea) and lower voicebox (larynx). Our intent is to implement all of the necessary steps for a successful new stem/progenitor cell-derived airway transplant for later stage clinical trials and/or commercialization within 4 years. Our team […]