Cell Lines of Dopaminergic Neurons Derived from Human Induced Pluripotent Stem Cell (iPSC) lines for Multiple Neurological Therapeutic and Diagnostic Uses

This technology includes three cell lines of dopaminergic neurons derived from human induced pluripotent stem cell (iPSC) line BC1, human iPSG line X1 and human embryonic stem cell (hESC) line H14 to be utilized in neurology research. These cell lines will be used for to study the biology of brain development and may also be used to test different characterization and differentiation assays. The dopaminergic neurons and/or their derivatives may also be used as controls in studies to screen for small molecules to change cell fate and/or to alleviate the phenotypes of various diseases.

Real-time Monitoring of In Vivo Free Radical Scavengers Through Hyperpolarized [1-13C] N-acetyl Cysteine as a Diagnostic and Disease Monitoring Tool

This technology includes synthesized demonstrated [1-13C] NAC as a promising novel probe for hyperpolarized 13C MRI methodologies which could provide diagnostic, and evaluation of response to treatment in various cancers and neurological diseases. N-acetyl cysteine (NAC) is a widely used therapeutic and involved to stimulate glutathione synthesis. Glutathione elevates detoxification and works directly as a free radical scavenger. In vivo hyperpolarized NAC was broadly distributed throughout the body.

Genetic Manipulation of Natural Killer Cells to Express c-MPL Growth Factor Receptor as a Therapy for Cancer

This technology includes genetic manipulation of natural killer (NK) cells to express thrombopoietin receptor (c-MPL) growth factor receptor as strategy to augment NK cell proliferation and anti-tumor immunity. Many investigational adoptive immunotherapy regimens utilizing NK cells require the administration of IL-2 or IL-15 cytokines to support the survival and function of the cells in patients, however administration of these cytokines causes a number of serious dose-dependent toxicities.

Blocking CD38 using Daratumumab F(ab)2 to Protect Natural Killer Cells from Daratumumab-induced Apoptosis and Cell Death for the Treatment of Multiple Myeloma

This technology includes the method of blocking CD38 in expanded natural killer (NK) cell therapy in combination with daratumumab in patients with multiple myeloma. Our in vitro studies have already confirmed the addition of NK cells to myeloma cells that have been exposed to daratumumab enhances myeloma killing compared to single agent treatment.

Fluorogen-binding RNA Aptamers for Imaging and Analysis of RNA

This technology includes a number of RNAs that can induce strong fluorescence of otherwise non-fluorescent small molecules to be used for imaging and analysis of RNA. These RNAs have many potential applications as tags for live-cell imaging of cellular RNAs, as well as reporters for in vitro diagnostics. The "Mango" family of fluorescent RNA-fluorophore complexes has been previously reported.

Single cell profiling of chromatin Occupancy and RNAs Sequencing (scPCOR-seq)

Cell-to-cell heterogeneity in gene expression is a widespread phenomenon, and may play important roles in cellular differentiation, function and disease development. Human Cell Atlas aims to profile gene expression in every single human cells. Recent studies have implicated a potential role of chromatin in the heterogeneity in gene expression. Understanding the mechanisms of cellular heterogeneity requires simultaneous measurement of RNA and occupancy of histone modifications and transcription factors on chromatin due to their critical roles in transcriptional regulation.

DLX3-floxed mice (DLX3f/f) for Use in Drug Development and In Vivo Research Studies for Ectodermal Dysplasia Disorders

This technology includes the creation of DLX3-floxed mice, specifically designed for conditional deletion of the DLX3 gene via Cre-mediated recombination. This innovative approach aims to develop mouse models for studying ectodermal dysplasia disorders. Ectodermal dysplasias are a diverse group of genetic conditions affecting the development of ectodermal structures, including hair, teeth, and bones. The DLX3f/f mice are particularly valuable for modeling specific disorders such as Tricho-dento-osseous syndrome (TDO), Amelogenesis Imperfecta (AI), and Dentinogenesis Imperfecta (DI).

Identification of a Novel Parvovirus for Vaccine Development and Use as a Diagnostic Tool

This technology includes a procedure for novel virus identification in a variety of human specimens by solexa high-throughput sequencing, which allows for the screening a large number of clinical specimens for novel virus discovery in a highly efficient and relatively economical method. By using this technique, we have successfully identified a novel parvovirus from samples of seronegative hepatitis patients.

Generation of AAVS1 and C13 “Safe Harbor” Transcription Activator-life Effector Nucleases (TALENs) for Drug Screening or Gene Therapy Development

This technology includes AAVS1 and C13 “safe harbor” transcription activator-life effector nucleases (TALENs) for drug screening or gene therapy applications. TALENs are engineered sequence-specific DNA endonucleases that can significantly enhance genome-editing efficiency by >100-1000 folds. “Safe harbor” such as AAVS1 safe harbor and C13 safe harbor is genome locus that allows robust and persistent transgene expression with no or minimal interference of endogenous gene expression and cell properties.