Disease Focus: Infectious Disease
A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering
Research Objective We propose to discover genome- and epigenome-edited allogeneic T cells engineered to selectively target JCV as a potentially lifesaving treatment for progressive multifocal leukoencephalopathy (PML). Impact If successful, we would revolutionize treatment for PML patients in whom T cell immunity cannot be restored or boosted and unable to wait for adoptive transfer of […]
An hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies.
Research Objective We propose to transduce hematopoietic stem cells with vectors that encode chimeric antigen receptors targeting HIV for T cells and anti-HIV broadly neutralizing antibodies for B and/or plasma cells. Impact Recent methods are limited by the rise of escape mutants against a single CAR. Our approach solves this issue by the ability to […]
Combating COVID-19 using human PSC-derived NK cells
Research Objective We propose to generate NK cells with enhanced immunity from gene-edited human PSCs and use the resultant NK cells to kill SARS-CoV-2-infected cells to combat against COVID-19. Impact The use of gene-edited hPSCs as a source for genetically engineered NK cells will allow us to generate effective immunotherapy for COVID-19 that has no […]
Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids
Research Objective To develop a new therapy for COVID-19 using human iPSC-derived lung organoids that targets SARS-CoV-2 protease known as the virus’ “Achilles Heel” Impact Our work, if successful, will bring a class of new drugs directly targeting viral enzyme and open the door for future COVID therapies. Major Proposed Activities Complete synthesis and testing […]
Engineering Lifelong Cellular Immunity to HIV
Research Objective We aim to uncover a therapeutic approach to attempt to treat and potentially cure HIV infection using gene modified blood forming stem cells to enhance the immune response against HIV. Impact The study will allow a potentially curative treatment for HIV infection, which currently doesn’t exist. This will eliminate the need to administer […]
Immunization strategies to prevent Zika viral congenital eye and brain disease
Research Objective Our objective is to utilize human iPSC-derived neural and ocular cells to identify growth attenuated and non-pathogenic Zika virus vaccine candidates that can prevent congenital ZIKV disease. Impact Currently, there are no therapies or vaccines available against ZIKV for human use. The human iPSC technology provides a unique opportunity to test the growth […]
A treatment for Zika virus infection and neuroprotection efficacy
Research Objective We propose to determine the impact of the Zika virus during human neurodevelopment and to test a FDA-approved therapeutic candidate to treat Zika infection. Impact A drug to treat/cure Zika infection and for neuroprotection. Major Proposed Activities To determine the molecular and cellular alterations caused by the Zika virus in the human developing […]
Immunotherapy for HIV infection using engineered hematopoietic stem/progenitor cells
Research Objective The therapeutic candidate proposed here is hematopoietic stem/progenitor cells engineered to encode for HIV-specific T cell receptors. Impact The success of the proposed studies will test the efficacy of an approach to provide long-lasting functional cure for HIV infection, obviating the need for anti-retroviral therapy. Major Proposed Activities Test if engineered hematopoietic stem/progenitor […]
Hematopoietic Stem/Progenitor Cell-Based Chimeric Antigen Receptor Gene Therapy for HIV Infection
Translational Candidate A blood forming stem cell based therapy to treat HIV infection and enhance HIV immunity. Area of Impact We are seeking to develop a therapy to treat HIV infection to replace standard drug therapy and cure people of the virus. Mechanism of Action We are seeking to develop a gene therapy that modifies […]
Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients
Translational Candidate Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy Area of Impact HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus Mechanism of Action The candidate oligonucleotide therapy, FD-86 disrupts viral IE feedback circuitry and breaks homeostatic control of cytotoxic IE proteins inducing apoptosis only in infected cells. Unmet Medical Need Treatment-resistant CMV […]