Therapeutic/Technology: Therapeutic Approach


Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs

Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum Disorder (ASD). Impact Our goal is to develop a small molecule to treat Autism Spectrum Disorder (ASD), which currently affects 1/68 children born in the […]

Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas

Research Objective We will develop a non-viral gene editing technology to replace the endogenous TCRα locus of stem memory T cells with transgene TCRs that are specific to brain cancer neoantigens. Impact Gliomas are lethal tumors often affecting children and young adults. Therapy using Tscm directed to attack truncal neoantigens in these tumors may provide […]

Universal Pluripotent Liver Failure Therapy (UPLiFT)

Research Objective Universal Pluripotent Liver Failure Therapy (UPLiFT) is composed of 2 lines- UPLiFT0 ( from LiPSC-GR1.1) and UPLiFT1 which will be derived from gene edited universal human pluripotent stem cells. Impact In some liver-based metabolic diseases, replacement of 5-10% of the liver mass may salvage the patient. Transplantation of hepatic progenitors from universal donor […]

Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases

Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new treatments for achromatopsia and cone-rod dystrophy. These are rare hereditary blinding diseases with no cures Major Proposed Activities Transcriptomic and proteomic profiling of control and […]

Generation and in vitro profiling of neural stem cell lines to predict in vivo efficacy for chronic cervical spinal cord injury.

Research Objective This project generates new cGMP compliant tissue educated human neural stem cell lines, paired with in vivo pre-clinical proof of concept testing, and development of a predictive in vitro profile. Impact Identification of new cell lines with in vivo efficacy testing to enable efficient translation to chronic cervical spinal cord injury, an area […]

Silicon Nanopore Membrane encapsulated enriched-Beta Clusters for Type 1 Diabetes treatment

Research Objective We propose to develop a cell encapsulation technology to support the long term viability and function of human stem cell derived insulin producing cells. Impact A device that provides adequate mass transfer of oxygen, glucose, and insulin for encapsulated stem cell derived beta cells can address the challenges of current cell therapy for […]

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 […]

Targeting Cancer Stem Cells in Hematologic Malignancies

Research Objective We will develop a biotherapeutic/monoclonal antibody that blocks the growth of human AML cancer stem cells in vitro and in vivo. Impact Treatment of the cancer stem cell driven disease Acute Myelogenous Leukemia (AML) will be impacted. AML is the most common acute leukemia in adults and current treatments are largely ineffective. Major […]

iPS Glial Therapy for White Matter Stroke and Vascular Dementia

Research Objective This cell line will target recovery in ischemic white disease, a progressive dementing condition with no current therapy by developing a new stem line, iPS-glial enriched progenitors (iPS-GEPs). Impact This cell line will target tissue repair and recovery in ischemic white disease/vascular dementia, a chronically progressive and dementing condition with no current therapy. […]

Identification and Generation of Long Term Repopulating Human Muscle Stem Cells from Human Pluripotent Stem Cells

Research Objective We will molecularly and functionally define muscle stem cells in human muscle in development, juvenile and adult and develop strategies to generate the most regenerative muscle stem cells from hPSCs. Impact There is no clinically relevant cell endowed with continuous repopulation ability from hPSCs. This work could provide a cell therapy for muscle […]

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