LRRK2 Inhibitors: Novel Treatment for Intestinal Bowel Disorders

Use of Leucine Rich Repeat Kinase 2 (LRRK2) inhibitors for the treatment of Intestinal Bowel Disorders (IBD) is disclosed. IBD is a broad term that describes conditions with chronic or recurring immune response and inflammation of the gastrointestinal tract. Crohn's disease and ulcerative colitis, two common forms of idiopathic IBD, are chronic, relapsing inflammatory disorders of the gastrointestinal tract.

Highly Sensitive Tethered-Bead Immune Sandwich Assay

This technology is a highly sensitive tethered-bead immune sandwich assay. Analyte molecules are captured between two antibodies, a capture antibody and a detection antibody. The capture antibody on a micron-size bead binds analyte from a sample fluid. The bead-captured analyte is then exposed to a “detection” antibody that binds to the bead-captured analyte, forming a “sandwich”. The sandwiched analyte-bead complex then connects to a flexible polymer (such as DNA) anchored on a solid surface to form tethered particles.

CXCR4 Reduction Leads to Enhancement of Engraftment of Hematopoietic Stem Cells

Methods of enhancing engraftment of donor hematopoietic stem cells (HSCs) by reducing expression or activity of CXCR4 in HSCs is described. HSC are the only cells in the bone marrow that are both pluripotent and long lived. Bone marrow transplantation (BMT) using HSC is an increasingly common medical therapy for severe hematologic cancers and primary hematologic immunodeficiencies. However, for significant HSC engraftment to occur there must usually be pre-transplant conditioning with either irradiation or chemotherapy or both.

T Cell-Based Adoptive Transfer Immunotherapy for Polyomavirus-Associated Pathologies

Available for licensing are methods to generate T cells responsive to multiple polyomaviruses. The resulting T cell populations could be useful in treating immunosuppressed individuals with polyomavirus infections or polyomavirus-associated pathologies such as Merkel cell carcinoma (MCC), polyomavirus-associated nephropathy (PVAN), hemorrhagic cystitis, progressive multifocal leukoencephalopathy (PML), and trichodysplasia spinulosa (TS). The methods could also be used to restore polyomavirus-specific immunity in immunocompromised individuals.

A Novel Therapeutic Vector for Hemoglobin Disorders

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector.

Real-time RT-PCR assay for Detection of Live Attenuated Influenza Vaccine for A and B Viruses

Upon intranasal vaccination, live attenuated influenza vaccine (LAIV) viruses may replicate within the nose for several days. Current clinical diagnostic tests cannot distinguish between LAIV viruses and multiple influenza viruses in recently inoculated patients that present with respiratory symptoms. This poses a problem for the diagnosis and treatment of patients with respiratory symptoms, as these symptoms may not be caused by influenza. CDC researchers have developed a real-time RT-PCR assay to detect the presence of LAIV viruses.

SIRT1 KO Human Cell Lines Generated by CRISPR/Cas9-mediated DNA Editing

SIRT1, a NAD+-dependent protein deacetylase, is the most conserved member of the sirtuins family. Through deacetylation of a number of protein substrates that are important transcription factors or co-factors, SIRT1 regulates many vital biological processes such as metabolism, cellular stress response, stem cell pluripotency, and development.

Mouse Model for Study of Diabetic Nephropathy and Role of Soluble Epoxide Hydrolase

Diabetic nephropathy (DN) is the leading cause of renal failure and is characterized by proteinuria that progresses to renal inflammation and decline in the glornerular filtration barrier (GFB). Podocytes are specialized epithelia cells in the glomerular capsule that have a role in filtration of blood and maintaining the integrity of the GFB; dysfunction of these cells plays a significant role in the pathogenesis of DN. Soluble epoxide hydrolase (sEH) is a cytosolic enzyme whose inhibition has beneficial effects in inflammatory diseases.

Remotely Monitored Mouse Feeding Experimentation Device

How much does a mouse eat per day? If a researcher is conducting dietary studies, the answer is very important. For instance, obesity studies require accurate measures of feeding. Existing automated methods for taking feeding measurements are expensive and use specialized caging that is not compatible with typical vivarium colony racks. As a result, many researchers simply weigh food each day or two to determine how much food the mice ate. This is time-consuming, can be error prone, and provides a low temporal resolution view of feeding.

Bag6 Polyclonal Antibodies That Recognize Human Bag6 Protein

Bag6 (BCL2 associated athanogene 6) is a multifunctional chaperone involved in tail anchored protein biogenesis, endoplasmic reticulum-associated protein degradation, and degradation of mislocalized membrane proteins. It is the central component of a stable three chaperone complex that also contains two cofactors-Ubl4A and Trc35. This complex acts in conjunction with the co-chaperone SGTA to channel proteins bearing an exposed hydrophobic segment in the cytosol to avoid protein aggregation.