A First-in-Class Treatment for Progressive Multifocal Leukoencephalopathy Via Multimodal Immune System Engineering

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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,…

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An hematopoietic stem-cell-based approach to treat HIV employing CAR T cells and anti-HIV broadly neutralizing antibodies.

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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…

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Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

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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,…

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