Disease Focus: Infectious Disease
Stem cell differentiation to thymic epithelium for inducing tolerance to stem cells
The thymus is an organ that plays a key role in controlling immune responses and immune tolerance. The thymus promotes immune tolerance by deleting and removing self-reactive T cells from the immune system. In addition, the thymus also helps drive the production of important suppressor T cell populations like regulatory T cells that also control […]
Human Embryonic Stem Cell Therapeutic Strategies to Target HIV Disease
AIDS is a disease that currently has no cure. It arises when the human immunodeficiency virus (HIV) infects certain types of blood cells. These cells would normally be used to fight infection, but instead are destroyed by the virus, leading to immunodeficiency. We have recently been able to induce the development of human embryonic stem […]
Genetic modification of the human genome to resist HIV-1 infection and/or disease progression
The proposed studies describe the genetic approaches utilizing human embryonic stem cells to suppress and/or eliminate the expression of the human protein CCR5. CCR5 is found on the surface of white blood cells. HIV-1 attaches to CCR5 and uses CCR5 to enter into its target cells. Our approach is to utilize established as well as […]
GENE-MODIFIED HEMATOPOIETIC STEM/PROGENITOR CELL BASED THERAPY FOR HIV DISEASE
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 reproduction within the body. When RNA interference is introduced into a stem cell, its blocking activity will be present throughout the lifetime of the stem […]
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 to a cell. Building on this observation, a study published in 2009 reported a potential “cure” in an AIDS patient with leukemia after receiving a […]
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. Impact Neurodevelopmental disorders caused by immune cell perturbations will be impacted by the fundamental understanding of how immune system controls normal development of the brain. […]
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 translational discoveries. Impact Our work will identify the biological factors that influence inter-individual differences in susceptibility and immune response to neurotropic viruses, which could inform […]
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 the siRNA delivery problem and rapidly open the door to Anti-COVID siRNA therapeutics. Major Proposed Activities Complete synthesis of a new delivery device called a […]
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, cough, or positive disease-related chest x-ray. Therapeutic Mechanism CYNK-001 is allogenic, human placental hematopoietic stem cell-derived NK cells that express the dominant NK cells marker […]