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…
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…
Therapeutic Candidate or Device Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis. Indication The proposed studies provide an innovative stem cell based approach with gene…
The lung, along with the skin and gut are the three organs in perpetual contact with our environment. The lung has evolved mechanisms for repairing injury due to exogenous noxious…
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…
Idiopathic Pulmonary Fibrosis (IPF) is a progressive and generally fatal disease that causes scarring of the lungs and therefore an inability to breathe. Its true prevalence is unknown, as it…
The primary goal is to bring a safe and effective therapy to children with severe large airway disease. Our intent is to implement all of the necessary steps for a…
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