Codon-Optimized Gene Therapy for Niemann-Pick Disease Type C

Niemann Pick Disease Type C (NPC) is a rare and fatal, autosomal recessive, neurodegenerative disease that can present in infants, children, or adults. Most patients with NPC have mutations in NPC1, a gene implicated in intracellular cholesterol trafficking, which results in intracellular accumulation of unesterified cholesterol in late edosomal/lysosomal structures and of glycosphingolipids, especially in neuronal tissue. No curative therapy exists at present.

Structure-Based Design of SARS-CoV-2 Spike Immunogens Stabilized in the RBD-All Down Conformation

SARS-CoV-2 has emerged as a global pathogen, sparking urgent vaccine development efforts. The trimeric SARS-CoV-2 spike appears to be a leading vaccine antigen. However, the inability of antibodies such as CR3022, which binds tightly to a cryptic spike epitope, to neutralize SARS-CoV-2 suggests a spike-based means of neutralization escape.

Identification of a New Human Monoclonal Antibody that More Potently Prevents Malaria Infection

Malaria is a major disease caused by a parasite transmitted through the bite of infected female mosquitoes. Globally, an estimated 214 million cases of malaria and 438,000 deaths from malaria occur annually, with chidren in African and South Asian regions being most vulnerable. Approximately 1,500-2,000 cases of malaria are reported in the United States each year, mostly in returning travelers from malaria- endemic countries.

Prefusion-Stabilized Fusion (F) Glycoprotein Vaccine Immunogens For Human Metapneumovirus

Human metapneumovirus (hMPV) infections have been shown as a common cause of upper and lower respiratory diseases such as bronchiolitis and pneumonia in young children, the elderly, and other immunocompromised individuals. Studies show that infections by the non-segmented negative strand RNA virus begin with attachment and entry of viral glycoproteins that mediate fusion with host cellular membranes. Like for the human respiratory syncytial virus (hRSV), a viral entry is initiated by the fusion (F) protein.

A VSV-EBOV-Based Vaccine Against COVID-19

Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is the causative agent of for coronavirus disease 2019 (COVID-19). COVID-19 is characterized by fever, cough, difficulty breathing, loss of taste and smell, nausea, and sore throat. As of the fourth quarter 2020, COVID-19 is responsible for over 1.17 million deaths worldwide. As the pandemic continues to surge, the importance of a safe, affordable, and efficacious vaccine is of urgent importance.

Newcastle Disease Virus-Like Particle Displaying Prefusion Stabilized SARS-CoV-2 Spike and Its Use

SARS-CoV-2 has resulted in a global pandemic, sparking urgent vaccine development efforts. The trimeric SARS-CoV-2 spike stabilized in its prefusion conformation by the addition of 2 proline mutations (“SARS-CoV-2 S2P”) is the antigenic basis of SARS-CoV-2 vaccines that are currently authorized for use in the United States.

Hybridomas to Human Immunoglobulins for SARS-CoV-2 Diagnostics and Additional Indications

Immunoglobulins play a key role in the immune system. CDC has developed and tested hybridoma cell lines (monoclonal antibody (mAb) clones) for human IgG and other immunoglobulins. The mAbs generated from those hybridomas could be used as a reagent (second Ab) of anti-human immunoglobins in a diagnostic assay for SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2), the virus that causes COVID-19 (coronavirus disease 2019) and other assays that detect antigen specific antibodies from human sera.

Diagnostic Assay to Detect Group C Rotavirus in Humans and Animals—Monoclonal Antibody-based ELISA (Enzyme-linked Immunosorbent Assay)

Rotaviruses cause severe gastroenteritis in humans and animals globally. Currently, there are eight known serogroups (A-H) of rotaviruses. Group C rotavirus (GpC RV) causes sporadic cases and outbreaks of acute diarrhea in children and adults worldwide. GpC RV is also associated with diarrhea in swine. Currently, no simple and reliable diagnostic test exists for GpC RV, so disease prevalence remains unknown.

Genetic Polymorphisms Of Interleukin-1 Alpha And Beta Associated With Early Onset Periodontitis

Periodontal disease occurs in 10-20% of adults, and constitutes a major cause of tooth loss. About 0.5% of U.S. adolescents between the ages of 14 to 17 years old (about 70,000) have localized early onset periodontitis and 0.1% (17,000) have the more destructive form known as generalized early onset periodontitis. Both types of early onset periodontitis often lead to tooth loss before the age of 20. Extrapolation of these figures up to age 35 leads to estimates of early onset periodontitis having a major impact on the dental health of 400,000 individuals in the U.S. population.

Tumor Associated Calcium Signal Transducer 2 (TACSTD2)-Overexpressing Huh7.5 Cells That Are More Permissive to HCV Cell Entry and Replication Compared to the Model Huh7.5 Cell Line

Worldwide, 130-150 million individuals are chronically infected with hepatitis C virus (HCV), a major cause of liver-associated morbidity and mortality worldwide. Despite recent advances in antiviral drugs that can cure some individuals, a rapid decline of the global disease burden is hampered by remarkably high treatment costs and a high number of undiagnosed infections. Moreover, a significant number of patients develop resistance and additional treatment modalities may be needed to dramatically reduce the worldwide incidence of HCV infection.