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…
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…
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…
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…
Research Objective This study examine potential immunologic changes caused by cellular reprogramming that could present a barrier to clinical application of regenerative therapies. Impact Identification and evaluation of immunologic changes…
Research Objective This project aims to develop a sensitive and non-invasive method for tracking stem cells in clinical trial, without the need for genetically engineered reporters or long-lived radioisotopes. Impact…
Translational Candidate Human stem cells in a dish engineered into heart cells to supplement, refine, reduce, and/or ultimately replace human clinical trials. Area of Impact Increase genetic diversity of preclinical…
Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and…
Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA)…
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