Novel Methods for Generating Retinal Pigment Epithelium Cells from Induced Pluripotent Stem Cells

The retinal pigment epithelial cells (RPE) make up a polarized monolayer in the vertebrate eye that separates the neural retina from the choroid, and performs a crucial role in retinal physiology by forming a blood-retinal barrier and closely interacting with photoreceptors to maintain visual function.  Many ophthalmic diseases, such as age-related macular degeneration, are associated with a degeneration or deterioration of the RPE. 

Establishment of Induced Pluripotent Stem Cells (iPSC) from the Thirteen-lined Ground Squirrel

The limited choice in cell types available for in vitro studies has become an obstacle in hibernation research. 

Researchers at the National Eye Institute for the first time have successfully established iPSC line(s) from a mammalian hibernator, which can be potentially used to generate various cell types and tissue models for in-depth mechanistic studies of hibernation and coldness tolerance in vitro. 

Novel Dopamine D2 Receptor Antagonists and Methods of Their Use

Investigators at the NIH have identified a series of novel, small molecule antagonists of the dopamine D2 receptor. Among the dopamine receptor (DAR) subtypes, D2 DAR is arguably one of the most validated drug targets in neurology and psychiatry. For instance, all receptor-based anti-Parkinsonian drugs work via stimulating the D2 DAR, whereas all FDA approved antipsychotic agents are antagonists of this receptor. Unfortunately, most agents that act as antagonists of D2 DAR are problematic, either they are less efficacious than desired or cause multiple adverse effects.

Personalized Tumor Vaccine and Use Thereof for Cancer Immunotherapy

Immune checkpoint inhibitors (ICIs) vastly improved the outcome of various advanced cancers; however, many are less likely to respond to single-agent ICI. Tumors with low T-cell infiltration are "immunologically cold" and less likely to respond to single-agent ICI therapy. This diminished response is presumably due to the lack of neoantigens necessary to activate an adaptive immune response. On the other hand, an "immunologically hot" tumor with high T-cell infiltration has an active anti-tumor immune response following ICI treatment.

A Neural Stem Line from a Niemann Pick C (NPC) Type 1 Patient for Therapy Development

This technology includes a neural stem cell (NSC) line derived from a Niemann Pick C (NPC) patient, aimed at advancing research and drug development for NPC, an inherited neurodegenerative disorder characterized by the accumulation of cholesterol in neurons. The NSCs, which serve as a crucial intermediate cell type, can be differentiated into any neuronal or glial cell of the brain or spinal cord under appropriate culture conditions. These cells originate from fibroblasts reprogrammed into induced pluripotent stem cells.

Neural Stem Cells from an iPSC Line Ubiquitously Expressing Green Fluorescent Protein for Basic Science Research and Cell Line Tracking

This technology involves neural stem cells (NSCs) derived from pluripotent stem cells (PSCs) that can differentiate into neurons and glia. The key feature of this technology is the CY2 EEF1A1 GFP iPSC line, which includes a green fluorescent protein (GFP) expressed under the EEF1A1 promoter, leading to its ubiquitous expression in cells. This characteristic makes the NSCs and the neural cells differentiated from this line exhibit green fluorescence. Such cells, when transplanted into animal models like mice and rats, can be easily tracked due to their fluorescence.

Conditional Cell Immortalization Plasmid for Basic Science Research

This technology includes a novel plasmid design for cell immortalization. It uniquely combines the conditional activation of human telomerase and c-myc genes through cumate addition, a method distinct from traditional immortalization techniques which commonly use SV40 T-antigen, telomerase, or c-myc. This plasmid also includes a GFP reporter and a puromycin resistance gene, enhancing the efficiency of the immortalization process.

Neuronal Differentiation of Neural Stem Cells with StemPro Embryonic Stem Cell Serum Free Medium for Research and Therapeutic Development

This technology involves an innovative method for differentiating neural stem cells (NSCs) into neurons, primarily for use in basic science research and in developing therapies for brain and spinal cord disorders. Existing methods for generating neurons from NSCs typically result in high efficiency but low survival rates, especially when neurons are dissociated and regrown. This new method utilizes Life Technologies StemPro embryonic stem cell serum-free medium, which significantly enhances differentiation efficiency into neurons with minimal cell death.

Oral Iron-Chelator Therapy for Treating Developmental Stuttering

This technology discloses the use of small-molecule iron chelators—drugs that bind and remove excess iron—for the oral treatment of developmental stuttering in children and adults. Mouse models carrying human stuttering mutations show both elevated striatal iron and impaired vocalization; daily low-dose deferiprone reverses these speech-like deficits while normalizing brain-iron MRI signals.