Capsid-Free AAV Vectors for Gene Delivery and Their Use for Gene Therapy

The invention concerns novel capsid-free AAV vectors that can be used for gene delivery and gene therapy applications. The invention provides for a linear nucleic acid molecule comprising in this order: a first adeno-associated virus (AAV) inverted terminal repeat (ITR), a nucleotide sequence of interest, and a second AAV ITR, wherein said nucleic acid molecule is devoid of AAV capsid protein coding sequences. The said nucleic acid molecule can be applied to a host at repetition without eliciting an immune response.

Transgenic Mice Expressing CNO-sensitive Gq- or Gs-coupled Designer Receptors Selectively in Pancreatic Beta Cells

Impaired functioning of pancreatic beta cells is a key hallmark of type 2 diabetes. Beta cell function is modulated by the actions of different classes of heterotrimeric G proteins. The functional consequences of activating specific beta cell G protein signaling pathways in vivo are not well understood at present, primarily due to the fact that beta cell G protein-coupled receptors (GPCRs) are also expressed by many other tissues.

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.

Efficient mRNA-Based Genetic Engineering of Human NK Cells with High-Affinity CD16 and CCR7

A highly efficient method to genetically modify natural killer (NK) cells to induce expression of high affinity CD16 (HA-CD16) through mRNA electroporation, to potentiate NK cell-mediated antibody-dependent cellular cytotoxicity (ADCC). ADCC is mediated by CD16+ NK cells following adoptive NK cell transfer, but most humans express CD16 which has a relatively low affinity for IgG1 antibodies.

Pyrophosphate Analog HIV-1 Reverse Transcriptase Inhibitors

The invention relates to compounds that inhibit HIV-1 DNA synthesis mediated by reverse transcriptase (RT). HIV-1 DNA synthesis by RT utilizes deoxynucleoside 5’-triphosphate (dNTP) as substrate and like many other enzymes, the reaction is reversible. Pyrophosphate analogs like imidodiphosphate strongly promote reverse reaction dNTP products containing the imidodiphosphate group instead of the naturally occurring pyrophosphate group. This imidodiphosphate-containing dNTP was found to be a potent inhibitor of the forward RT reaction.

Development of a Transferrable Norwalk Virus Epitope and Detector Monoclonal Antibody

Noroviruses are now recognized as the major cause of non-bacterial gastroenteritis in all age groups, and efforts are underway to develop an effective vaccine. The lack of a robust cell culture system for human noroviruses has complicated vaccine development. Hence, norovirus virus like particles (VLPs) have played an important role in the understanding of virus structure, immune response, antigenic diversity, and vaccine design.

Novel Magnetic Resonance Spectroscopy (MRS) Technique to Quantify Brain Metabolites

With respect to quantification of metabolites in the brain, conventional methods of magnetic resonance spectroscopy (MRS) yield results that are highly variable and highly dependent on the sequence type being applied. This invention describes a novel MRS technique that involves preparing longitudinal steady states at different flip angles using trains of RF pulses interspersed with field gradients to quantify metabolites.

Novel Peptide of <em>Streptococcus pneumoniae</em> Surface Adhesion A (PsaA) Protein Associated with Adherence and Uses Thereof – for Vaccine Candidate, Therapeutic and Diagnostic Development

Streptococcus pneumoniae (S. pneumonia), bacteria commonly referred to as pneumococcus, are a significant cause of disease resulting in 1.5 million deaths every year worldwide according to the World Health Organization. The major types of pneumococcal disease are pneumonia (lung infection), bacteremia (bloodstream infection), and meningitis (infection of the tissue covering of the brain and spinal cord). Less severe pneumococcal illnesses include ear and sinus infections.

New Anti-Influenza Virus Neuraminidase 9 (N9) Monoclonal Antibody – for Prevention or Treatment of H7N9 Influenza (Flu) A with Less Likelihood of Drug Resistance

H7N9 influenza viruses are predominately avian (bird) pathogens, however, since 2013, they have infected more than 1500 humans with a mortality rate of nearly 40% in confirmed cases. H7N9 viruses continue to be a threat to public health. Treatment for people infected with H7N9-subtype influenza A (H7N9) commonly includes the use of drugs that inhibit neuraminidase, a protein found on the virus’ surface. However, like other influenza viruses, H7N9 can become resistant to these drugs.

A Novel Reagent for Labeling PET Tracers at Trifluoromethyl Groups

The molecular imaging technique of positron emission tomography (PET) is an increasingly important tool in biomedical research and in drug discovery and development. Many small molecule drugs and potential PET radiotracers carry trifluoromethyl (CF3) groups. Because CF3 groups are generally considered to be metabolically stable, there is a strong interest in developing drugs with these groups.