Radiotherapy and Imaging Agent-based on Peptide Conjugated to Novel Evans Blue Derivatives with Long Half-life and High Accumulation in Target Tissue

This technology includes a newly designed, truncated Evans Blue (EB) form which allows labeling with metal isotopes for nuclear imaging and radiotherapy. Unlike previous designs, this new form of truncated EB confers site specific mono-labeling of desired molecules. The newly designed truncated EB form can be conjugated to various molecules including small molecules, peptides, proteins and aptamers to improve blood half-life and tumor uptake, and confer better imaging, therapy and radiotherapy.

A Novel Therapy/Companion Diagnostic (BAM15 And mtDNA) for Sepsis and Sepsis-induced Acute Kidney Injury

This technology includes a therapy and companion diagnostic which can be used for the early diagnosis and treatment of sepsis and sepsis-induced acute kidney injury (AKI). Mitochondrial damage plays a key role in sepsis-induced acute kidney injury BAM15 [2-ftuorophenyl){6-[(2- fluorophenyl)am ino]{1 ,2,5-oxadiazolo[3,4-e]pyrazin-5-yl)}amine] is a mitochondrial uncoupler that protects mitochondria with more specificity and less cytotoxicity than other uncouplers. Mitochondrial DNA (mtDNA) is a damage associated molecular pattern that is increased in human sepsis.

RNASEH-Assisted Detection Assay for RNA

Several viral epidemics – such as the epidemics caused by H1N1 influenza virus, human immunodeficiency virus (HIV), Ebola virus, Zika virus, severe acute respiratory syndrome (SARS) virus, Middle East respiratory syndrome (MERS) virus and SARS-CoV-2 – have profoundly impacted global human health. Early identification of infected and/or infectious persons and isolating them from the population are some of the most effective and evident measures to prevent human-to-human spreading.

Methods of Treating Diffuse Large B Cell Lymphoma Based on Particular Genetic Subtype (LymphGen) - A Genetic Classifier to Aid in the Molecular Diagnosis and Treatment of Diffuse Large B-cell Lymphoma

The development of precision medicine approaches for DLBCL (Diffuse Large B Cell Lymphoma) is complicated by its genetic, phenotypic and clinical heterogeneity. Current classification methods do not fully explain the observed differences in clinical outcomes to current chemotherapy and targeted therapy. Therefore, better analytical methods to classify and predict DLBCL patients’ treatment response are needed.

Clinical Outcome Predictors for Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a group of aggressive B-cell lymphomas displaying heterogeneous outcomes after treatment.  Some patients have the slowly progressing disease that does not require immediate treatment, while others have a disease that rapidly progresses despite highly aggressive treatment. A number of prognostic tools have been described to determine whether patients have slow or rapidly progressing diseases, including the mantle cell lymphoma International Prognostic Index (MIPI) and biomarkers, such as KI-67.

Modulating Autophagy as a Treatment for Lysosomal Storage Diseases

Researchers at NIAMS have developed a technology for treatment of lysosomal storage diseases by inhibition of autophagy. Pompe disease is an example of a genetic lysosomal storage disease caused by a reduction or absence of acid alpha-glucosidase (GAA). Patients with Pompe disease have a lysosomal buildup of glycogen in cardiac and skeletal muscle cells and severe cardiomyopathy and skeletal muscle myopathy. Treatment of Pompe disease by GAA enzyme replacement therapy is quite ineffective for the skeletal muscle myopathy.

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.

Engineered Human Induced Pluripotent Stell Cell (iPSC) Lines for Multiple Therapeutic and Diagnostic Uses

This technology includes ten engineered human induced pluripotent stem cell (iPSC) lines with reported genes inserted into safe harbor sites for use in therapy and diagnostic screening assay development as well as basic stem cell biology research. These cell lines have the potential to differentiate into all cells in the body, and theoretically can proliferate/self-renew indefinitely.

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.

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