Therapeutic/Technology: Therapeutic Approach


Identification and characterization of the optimal human neural stem cell line (hNSC) for the treatment of traumatic brain injury (TBI) 2.0.

Research Objective We propose to discover the optimal human neural stem cell candidate for traumatic brain injury. 4 hNSC products (2 ES derived & 2 fetal) will be compared with TBI/vehicle controls, & then each other. Impact Traumatic brain injury (TBI) affects more Americans than brain, breast, colon, lung and prostate cancer combined ! There […]

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 […]

Discovery of therapeutics for Huntington’s Disease

Research Objective The objective of the proposed research is to perform 3 independent hESC-based screens to identify drug candidates for Huntington’s Disease. Impact There are currently no effective treatments for HD. Combination of human isogenic HD-mutants, novel tools and technology will provide therapeutic solutions for this neurodegenerative orphan disease. Major Proposed Activities Screening of 2,000 […]

Targeted Gene Editing in the Treatment of X-Linked Hyper-IgM Syndrome

Research Objective We are seeking to develop site-specific hematopoietic stem cell gene therapy with autologous transplant as a definitive treatment option for X-linked Hyper-IgM Syndrome. Impact These studies would bring stem cell gene therapy for X-HIGM closer to the clinic, as there are currently no options for those without an HLA match or with infections […]

Microenvironment for hiPSC-derived pacemaking cardiomyocytes

Research Objective This proposal investigates the effects of the microenvironment on the development and maintenance of pacemaking function in human induced pluripotent stem cell (hiPSC)-derived cardiomyocytes. Impact Pacemaking function of hiPSC-derived cardiomyocytes is lost over time. Sustainability of pacemaking function of these cells is critical for engineering an biopacemaker from the patient's own cells. Major […]

Multipotent Cardiovascular Progenitor Regeneration of the Myocardium after MI

Research Objective We developed technology to reproducibly prepare large numbers of bonafide cardiac progenitor cells from patient iPSCs. We propose the first test of these cells as a therapy for myocardial infarction. Impact Heart failure resulting from myocardial infarction is responsible for 13% of human mortality (WHO statistic). This proposed therapy is to restore the […]

A Novel Approach to Eradicate Cancer Stem Cells

Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]

A Novel Approach to Eradicate Cancer Stem Cells

Research Objective The outcome is a therapeutic candidate ready for Investigational New Drug (IND)-enabling studies to target a central hub of stemness pathways of cancer stem cells (CSC) maintenance and self-renewal Impact To date, the majority of metastatic cancers remain incurable, because CSCs that can grow new tumors evades current therapy. The proposed studies aim […]

Preclinical development of AAV vector-mediated in vivo hepatic reprogramming of myofibroblasts as a therapy for liver fibrosis

Research Objective An intravenously injectable virus that converts the scar cells responsible for liver cirrhosis into the cells that provide most of the liver’s function, thereby preventing or reversing liver failure. Impact The proposed research will develop a new therapy for liver cirrhosis, which can be cured by liver transplantation, but there are not enough […]

Lgr5-mediated self-renewal in B cell selection and leukemia-initiation

Research Objective LGR5-antibody drug conjugate to target LIC in B cell tumors that undergo self-renewal Impact LIC were only defined in myeloid leukemia, while LIC populations in B cell tumors remain elusive. LICs give rise to drug-resistance and relapse and remain unsolved clinical problems in B cell tumors. Major Proposed Activities Proof of concept studies- […]