Therapeutic/Technology: Biologic
Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis
Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS disease phenotypes Impact Our goal is to determine if ataxin-3 genetic dysregulation is a target for the development of therapies to treat ALS (Lou Gehrig's […]
Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use
Therapeutic Candidate or Device The therapeutic candidate is COVID-19 convalescent plasma (CCP) Indication The target indication is treatment of severe COVID-19 infection Therapeutic Mechanism Neutralizing antibodies are part of the humoral response of the adaptive immune system against viruses, and can be detected in plasma of convalescent individuals. Transfusion of COVID-19 convalescent plasma can provide […]
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 […]
Identifying HLA Class I Restricted Peptides That Induce CD8+ T Cells Against SARS-CoV-2
Research Objective A vaccine to help prevent COVID-19 Impact There is a clear need for a vaccine to prevent the spread of the COVID-19 coronavirus that is effective, can be rapidly produced and can be scaled for worldwide demand. Major Proposed Activities Identify structural regions of SARS-Cov-2 that can inhibit viral entry Identify potential regions […]
Biomaterial vaccine to enhance the formation of SARS-CoV-2-specific T memory stem cells
Research Objective The objective of this project is to develop an injectable biomaterial platform that can induce TMSCs and boost immunoactivation to vaccines against SARS-CoV-2, which will help protect elderly people. Impact This approach will boost T memory stem cell production to enhance immunization, and address the low/weak immunoresponses to vaccines, especially in the elderly […]
Human neural stem cell (hNSC) derived exosomes vs CSC14 hNSCs for the treatment of traumatic brain injury (TBI)
RNA-based therapeutics to augment regulatory T cells: a novel approach to treat myocarditis
Research Objective Use human cell therapy insights, specifically CDC-secreted EV analysis, to develop a noncoding RNA chemical entity for myocarditis treatment. Impact Key knowledge gap is how to recruit adaptive immunity to limit inflammation/heart injury in myocarditis. Boosting regulatory T cells is not yet a viable option. Major Proposed Activities Investigate the mechanism(s) by which […]
Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy
Research Objective A vaccine booster to induce antigen-specific T memory stem cells that will help enhance the long-term immunity against cancer recurrence Impact Cancer recurrence presents an unmet medical need. Cancer vaccines are promising, but often lack a long-term protection. We will induce T memory stem cells (TMSCs) to boost the long-term immunity. Major Proposed […]
Modified RNA-Based Gene Therapy for Cardiac Regeneration Through Cardiomyocyte Proliferation
Research Objective Efficacious and safe intramyocardial delivery of modified mRNA encoding cell cycle regulators as a gene therapy for cardiac regeneration through resident cardiomyocyte proliferation. Impact This project would provide disease-modifying gene therapy for people with heart failure due to loss of cardiac muscle, a leading cause of deaths in the US, using novel modified […]
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. […]