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…
Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome
Research Objective We propose a platform to generate transplantable thymus organoids derived from human pluripotent stem cells designed to treat severe immunodeficiencies in children affected by 22q11 DS Impact Our…
GENE EDITING FOR FOXP3 IN HUMAN HSC
Research Objective CRISPR/Cas9 mediated FOXP3 gene editing in patient-derived hematopoietic stem cells as a cure for IPEX syndrome Impact FOXP3 mutation in IPEX syndrome leads to immune system dysregulation. Allogeneic…
Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis
Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes…
Ex Vivo Gene Editing of Human Hematopoietic Stem Cells for the Treatment of X-Linked Hyper-IgM Syndrome
Translational Candidate Human hematopoietic stem cells that have been gene-corrected at the CD40L gene to treat patients with X-Linked Hyper-IgM Syndrome Area of Impact These studies will bring stem cell…
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