Proof of Concept – Page 3

Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

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Post published:November 28, 2025
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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…

The role of WNT and BMP signaling pathways in iPSC to iTenocyte step-wise differentiation for tendon repair

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Post published:November 28, 2025
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Research Objective Development-inspired differentiation will enable efficient and specific generation of tenocytes that can repair tendon injury, restore dysfunctional tissue, and prevent long term effects. Impact This study will eliminate…

Development of universal off-the-shelf iPSC derived dendritic cells for use as patient specific anti-tumor vaccine

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Post published:November 28, 2025
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Research Objective This proposal aims to develop a novel cell based vaccine for the treatment of cancer. The vaccine would be derived from stem cells and be personalized to the…

Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy

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Post published:November 28, 2025
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Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients…

Battling COVID-19 Using Off-The-Shelf HSC-Engineered iNKT Cells

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Post published:November 28, 2025
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Research Objective allogeneic HSC-engineered iNKT (HSC-iNKT) cells Impact treatment for COVID-19 Major Proposed Activities Milestone 1. Production of AlloHSC-iNKT and UHSC-iNKT cellsMilestone 2. Characterization of the AlloHSC-iNKT and UHSC-iNKT cellsMilestone…

Chimeric Antigen Receptor Targeting Spike Glycoprotein of SARS-cov2

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Post published:November 28, 2025
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Research Objective We expect to generate iPSC derived NK cells expressing a CAR against SARS-cov2 that could be used as an off-the-shelf therapy for COVID-19 Impact The proposed studies will…

Pro-healing biomaterial for treating lung inflammation associated with COVID-19

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Post published:November 28, 2025
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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…

Persistent Off-the-Shelf meACE2-CAR-IL-15 NK Cells Derived from CD34(+) Cord Blood Stem Cells to Prevent and Treat COVID-19

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Post published:November 28, 2025
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Research Objective To develop and characterize meACE-2-CAR-IL15 NK cells expressing a mutated ACE2 and IL-15, allowing specific killing of SARS-CoV-2-infected cells and long in vivo persistence of the engineered cells.…

Identifying HLA Class I Restricted Peptides That Induce CD8+ T Cells Against SARS-CoV-2

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Post published:November 28, 2025
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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…

Application of PGE2/BPV, a muscle stem cell targeting therapeutic, to the treatment of COVID-19 associated diaphragm atrophy

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Post published:November 28, 2025
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Research Objective Intramuscular delivery of 2 repurposed FDA approved drugs to activate diaphragm stem cells to augment regeneration and restore strength to COVID-19 patients with diaphragm atrophy from ventilation. Impact…

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