Conditionally Immortalized Human Podocyte Cell Lines

Podocytes, cells of the visceral epithelium in the kidneys, are a key component of the glomerular filtration barrier. Podocyte damage and loss contribute to the initiation of glomerular diseases. NIH investigators recently established long-term urinary cell cultures from two patients with focal segmental glomerulosclerosis and two healthy volunteers, via transformation with the thermosensitive SV40 large T antigen (U19tsA58) together with human telomerase (hTERT).

Construct for Tetracycline Inducible Podocyte Specific Gene Expression in Mice

The National Institutes of Health announces the generation of a construct by ligating 2.5kb human podocin promoter sequence to gene encoding reverse tetracycline-controlled transcriptional activator which enables tetracycline-inducible podocyte specific gene of interest expression with another construct consisting of tetracycline responsive element, minimal CMV promoter and gene of interest.

Truncated Methanocarba Adenosine Derivatives as A3 Adenosine Receptor Antagonists

Novel A3 adenosine antagonists available for licensing. A3 receptors are particularly highly expressed in inflammatory cells, making it a potentially desirable target for inflammatory diseases. This technology relates to highly specific antagonists and partial agonists of A3 adenosine receptors, which are negatively coupled to adenylate cyclase and have been broadly implicated in inflammation, cardiovascular disease, endocrine conditions and cancer. Further, A3 adenosine receptors have been implicated in asthma and glaucoma.

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying this genetic defect. It is caused by inherited genetic mutations in the gene that encodes GCase, which result in reduced activity of the enzyme.

Design and Biological Activity of Novel Stealth Polymeric Lipid Nanoparticles for Enhanced Delivery of Hydrophobic Photodynamic Therapy Drugs

Nanoparticles such as lipid-based nanoparticles (LNPs) represent a relatively new era of targeted drug delivery systems wherein these biocompatible particles can carry the drug(s) of interest to a specific tumor site. The new generation of nanoparticles, known as stealth nanoparticles, are engineered to have a coating of polyethylene glycol polymer (PEG) or other glycolipids that enable them to evade the immune system and have a longer circulation lifespan as well as improved bioavailability to diseased tissue and reduced non-specific toxicity.
 

In vitro Generation of an Autologous Thymic Organoid from Human Pluripotent Stem Cells

The thymus is an integral part of the adaptive immune system as it generates T cells. Its function diminishes rapidly as the body ages, leading to a compromise of the immune system in the elderly. Reconstitution of adaptive immunity through mass production of different T cell types is therefore a therapeutic need in immunocompromised populations. Furthermore, production of T cells with specific receptors targeting cancer cells is an important cancer immunotherapy approach.

Genetically Modified Hematopoietic Stem And Progenitor Cells (HSPCs) And Mesenchymal Cells As A Platform To Reduce Or Prevent Metastasis, Treat Autoimmune And Inflammatory Disorders, And Rebalance The Immune Milieu And Dysregulated Niches

Cancer cells can spread to various regions in the body in a process called metastasis which is associated with non-responsive to treatment and thus reduced survival. Identifying the markers of metastasis has been a major concern in the field of cancer diagnosis and therapy. Interestingly, research has shown that there is an increase in myeloid progenitors and myeloid cells at various stages of metastasis in an attempt by the immune system to  suppress cancer cells. This presents a promising technology for cancer immunotherapy.

Efficient Methods to Prepare Hematopoietic Progenitor Cells in vitro for Therapeutic Use

Hematopoietic progenitor cells (HPC) are multi-potent hematopoietic lineage cells that can differentiate into any type of blood cell, including but not limited to erythrocytes, T cells, B cells, and natural killer cells. As such, they have high therapeutic potential in the fields of regenerative medicine and cancer immunotherapy, especially when generated from patient-derived induced pluripotent stem cells (iPSC). Currently, the most efficient protocol to produce HPCs is co-culturing human iPSCs (hiPSC) with mouse stromal cells as a two-dimensional (2D) monolayer.

Autophagy Modulators For Use in Treating Cancer

Cancer cells can upregulate autophagy – cell destruction – as a response to chemotherapy. Investigators in Dr. Melvin DePamphilis’ laboratory at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) have shown that compounds identified by screening a library of compounds blocks autophagy in some cancer cells (e.g., melanoma) but are not toxic to normal cells. Cancer cells with mutations in the BRAF oncogene are especially dependent on autophagy. Treatment of cancer cells with the BRAF mutation can increase the efficacy of chemotherapy.

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