HPSC based therapy for HIV disease using RNAi to CCR5.
RNA interference is a naturally occurring means to block the function of genes in our body. We propose that RNA interference can be used to block HIV-1 infection and its…
ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry…
Neuroimmune interactions in the developing human brain
Research Objective This project will identify mechanisms by which immune cells regulate human brain development, and use stem cell derived models to identify pathological changes induced by congenital virus infection.…
Village-based identification of human risk factors for viral neuropathogenesis
Research Objective We will identify the risk factors underlying viral infections of the fetal brain using a novel human stem cell-based platform that has the potential to accelerate basic and…
Development of Anti-COVID RNAi Therapeutics Using Human iPSC-Derived Alveolar Epithelial Cells
Research Objective To optimize a new approach to deliver Anti-COVID siRNAs into human iPSC-derived lung cells that can selectively kill the COVID virus Impact Our proposal, if successful, will solve…
A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19
Therapeutic Candidate or Device Human placental hematopoietic stem cell derived natural killer cells (CYNK-001) Indication SARS-CoV-2 positive patients requiring hospital admission and have any 2 out of 3 symptoms: fever,…
Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)
Therapeutic Candidate or Device Novel testing of a cell based therapy (Mesenchymal Stromal Cells) for respiratory failure from ARDS. Indication COVID-19 positive or negative ARDS patients Therapeutic Mechanism It will…
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…
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit…
Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy
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…
