Histone deacetylase (“HDAC”)-selective inhibitors covalently bonded to a linker covalently bonded to an E3 ubiquitin ligase ligand, and salts thereof; pharmaceutical compositions containing them; ...
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The present disclosure relates provides compounds according to Formula I or Formula II as well as compositions including such compounds useful for the prevention of schistosomiasis infection and/or sw...
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Described herein are CRISPR/Cas9 constructs designed for the C-terminal truncation of human amyloid precursor protein (APP) as well as methods of making and using such a construct.
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Compounds of formula (I) or formula (II), compositions and methods useful for treating and/or preventing a fungal infections are provided. wherein the substituents are as defined in the appended claim...
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Modified influenza virus neuraminidases are described herein that improve viral replication, thus improving the yield of vaccine viruses. Expression of such modified neuraminidases by influenza virus...
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Disclosed herein are circular zymogens of RNase 1 that have a proteolytic cleavage site and are activated by a specific protease. These circular zymogens are useful for treatment of disorders that are...
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Provided herein are engineered non-catalytic, non-toxic tetanus toxin variants and methods of using such engineered tetanus toxin variants as low dose, protective vaccines that are non-toxic and more ...
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An isolated recombinant influenza virus is provided having PA, PB1, PB2, NP, NS, M, NA and HA viral segments, wherein the PB1 viral segment encodes a PB1 with a residue other than isoleucine at positi...
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FDA-qualified cell lines must be used for virus production for human vaccines. There are relatively few of these lines available and several (WI-38, MRC-5) used for virus production are becoming senes...
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Drug resistant pathogens continue to emerge as the rate of the development of new antimicrobials to combat them continues to decline. Alarmingly, slow-growing bacteria associated with dangerous and pr...
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PTX, DTX, rapamycin and selumetinib are potent chemotherapeutic agents useful in the treatment of a variety of cancers. Due to their limited water solubility, PTX and DTX are commonly formulated for p...
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Friedreich’s Ataxia (FRDA) is a neurodegenerative disorder without treatments to halt or reverse disease progression, affecting roughly 9,000 Americans. FRDA is caused by triplet expansion in the fi...
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Pompe disease is a rare autosomal recessive genetic disease (mutations from both parent carriers have to be inherited by the offspring to see symptoms) that typically affects children during developme...
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Cancer immunotherapy – a therapy that stimulates and employs the body’s own immune system – is one of the most promising new approaches for treating cancer. Despite its tremendous potential, it ...
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Voice impairment (dysphonia) affects an estimated 20 million people in the U.S., resulting in reduced quality of life and direct health care costs exceeding $11 billion per year. Between 60 and 80 per...
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Staphylococcus epidermidis is an important Gram-negative bacterial pathogen in hospital-acquired infections, and the most common cause of infections on indwelling medical devices. The costs related to...
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Currently there is only one well-studied and established tumor-targeting radioactive agent in pediatric oncology (131I-MIBG), which only targets neuroblastoma and other rare neuroendocrine tumors. It ...
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Polypeptides, such as enzymes, antibodies, hormones and cytokines, are useful therapeutic agents. However, routes for systemically introducing such polypeptides to a subject are limited. Oral administ...
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The epigenetic modifier SIRT6 is of the sirtuin class of proteins. In transgenic mice, increased SIRT6 expression lowers LDL and triglyceride levels, improves glucose tolerance and increases mitochond...
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Hyperlipidemia is a condition strongly associated with obesity and is an independent risk factor for the development of coronary heart disease. To help combat hyperlipidemia, the pharmaceutical indust...
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Currently, delivering drugs directly into the brain requires invasive surgery. This is because a tight cellular layer called the blood-brain barrier (BBB) not only prevents harmful substances from ent...
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Indole is one of the most abundant heterocycles in pharmaceuticals and bioactive natural products, such as indometacin, a non-steroid anti-inflammatory drug, and vincristine, a natural product used to...
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Prostate cancer is a significant health risk for men over the age of 50 and currently is a pervasive health threat with as many as 200,000 new cases diagnosed each year in the US alone. At present, t...
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The androgen receptor (AR) is a steroid hormone receptor that plays a crucial role in the development of normal prostate tissue, as well as in the progression of prostate cancer. Patients with metasta...
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Multiple sclerosis (MS) is a chronic inflammatory autoimmune disease affecting 2.5 million people worldwide. The disease is difficult to manage and there is no cure. The underlying mechanism of MS pro...
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