Gene Therapy for Treatment of CRX-Autosomal Dominant Retinopathies

Description of Technology:

Mutations in the cone rod homeobox (CRX) transcription factor lead to distinct retinopathy phenotypes, including early-onset vision impairment in dominant Leber congenital amaurosis (LCA). Adeno-Associated virus (AAV) vector-mediated delivery of a CRX cDNA under the control of a CRX promoter region partially restored photoreceptor phenotype and expression of phototransduction genes in an in vitro model of CRX-LCA.

Polyvalent Influenza Virus-Like Particles (VLPs) and Use as Vaccines

Influenza virus is a major public health concern, causing up to 500,000 deaths annually. The current strategy of reformulating vaccines annually against dominant circulating strains leads to variable protective efficacy and is unlikely to protect against novel influenza viruses with pandemic potential. Thus, there is a great need for a vaccine that provides “universal” protection against influenza viruses.

A Full-Length Infectious cDNA Clone of Zika Virus from the 2015 Epidemic in Brazil as a Genetic Platform for Studies of Virus-Host Interactions and Vaccine Development

An arthropod-borne virus, Zika virus (ZIKV), has recently emerged as a major human pathogen. Associated with complications during perinatal development and Guillain-Barré syndrome in adults, ZIKV raises new challenges for understanding the molecular determinants of flavivirus pathogenesis. This underscores the necessity for the development of a reverse genetic system based on an epidemic ZIKV strain. This technology relates to the generation and characterization in cell cultures of an infectious cDNA clone of ZIKV isolated from the 2015 epidemic in Brazil.

Optimizing RSV Infection Monitoring and High-Throughput Screening Through GFP Expression in the First-Gene Position of Respiratory Syncytial Virus (RSV) Strain A2

In this technology, researchers have engineered a modified version of Respiratory Syncytial Virus (RSV) strain A2 using reverse genetics to incorporate green fluorescent protein (GFP) into the first-gene position. This genetic modification allows for the efficient monitoring of RSV infection and the screening of potential chemical inhibitors. The GFP expression can be easily detected through fluorescence microscopy in live or fixed cells, providing a sensitive tool for both research and drug discovery.

Advancements in Postexposure Prophylaxis: Evaluating High-Potency Rabies-Neutralizing Monoclonal Antibodies

This technology represents a significant advancement in the field of rabies prevention, focusing on the development of highly potent rabies-neutralizing monoclonal antibodies (mAbs) for use in postexposure prophylaxis (PEP). With two mAbs, F2 and G5a, displaying exceptional neutralizing titers of 1154 and 3462 International Units (IUs) per milligram, respectively, these antibodies have the potential to offer enhanced protection against rabies when administered alongside rabies vaccines.

A Fundamental Tool for Efficient Recovery of RNA Viruses through Reverse Genetics

BSR T7/5 cells represent a foundational advancement in virology, offering a robust platform for the recovery of RNA viruses via reverse genetics. Established over 20 years ago, these cells have proven instrumental in the recovery of a wide array of RNA viruses, particularly those belonging to the mononegavirales order.

Construction of Recombinant Baculoviruses Carrying the Gene Encoding the Major Capsid Protein, VP1, From Calicivirus Strains (Including Norovirus Strains Toronto, Hawaii, Desert Shield, Snow Mountain, and MD145-12)

The noroviruses (known as "Norwalk-like viruses") are associated with an estimated 23,000,000 cases of acute gastroenteritis in the United States each year. Norovirus illness often occurs in outbreaks, affecting large numbers of individuals, illustrated recently by well-publicized reports of gastroenteritis outbreaks on several recreational cruise ships and in settings such as hospitals and schools. Norovirus disease is clearly important in terms of medical costs and missed workdays, and accumulating data support its emerging recognition as important agents of diarrhea-related morbidity.

Fatty Acid Derivatives and Their Use for the Treatment and Prevention of Autoimmune, Inflammatory, and Pain Disorders

The discovery and selection of suitable compounds for the treatment and prevention of autoimmune, inflammatory, and pain disorders is a significant challenge. Researchers at National Institute of Aging (NIA) mitigated this issue. They discovered and synthesized numerous novel fatty acid derivatives (novel small molecules) that may ameliorate these conditions and provide treatment options for these disorders. In a relevant rat model, the fatty acid derivatives developed by NIA demonstrated:

Novel Human Islet Amyloid Polypeptides as Alzheimer’s Disease Biomarkers and Inhibitors of Amyloid Formation

Description of Technology:

Over 34 million Americans are living with diabetes. An estimated 6.5 million Americans are living with Alzheimer’s disease (AD) and type 2 diabetes mellites (T2DM). Amyloidosis due to aggregation of amyloid-β is key pathogenic event in AD, whereas aggregation of mature islet amyloid polypeptide (IAPP37) in human islet leads to β-cell dysfunction. A hallmark feature of T2DM is the accumulation of islet amyloid polypeptide fibrils in pancreatic islets. Such accumulations form amyloid plaques and cause apoptosis of -cells of islets. 

Thalidomide Analogs that Inhibit Inflammation and Angiogenesis

Thalidomide and its close analogs (lenalidomide and pomalidomide) are widely used to treat a variety of diseases, such as multiple myeloma and other cancers as well as the symptoms of several inflammatory disorders. However, thalidomide is known for its teratogenic adverse effects when first clinically introduced in the 1950s, and is associated with drowsiness and peripheral neuropathy. Hence, there is intense interest to synthesize, identify and develop safer analogs. 

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