Modified AAV5 Vectors for Enhanced Transduction and Reduced Antibody Neutralization

Scientists at the NIH disclosed a mutated adeno-associated virus (AAV) serotype 5 by modifying sialic acid binding regions which mediate viral entry into host cells. Preliminary results from animal studies suggest that this modification can increase transduction by 3-4 folds in salivary glands and muscles, and can significantly decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV. Thus, the modified AAV5 vectors seem to be optimal for gene therapy.

A Novel Adeno-Associated Virus for Gene Therapy

Scientists at the NIH disclosed a novel adeno-associated virus (AAV) termed "44-9." AAV44-9 based vectors have high gene transfer activity in a number of cell types, including salivary gland cells, liver cells, and different types of neurons (e.g., cells of the cortex, olfactory bulb, and brain stem, and Purkinje cells of the cerebellum). These vectors can increase the transduction efficiency and decrease the potential of being neutralized by preexisting antibodies compared to the wild type AAV.

Engineering Neural Stem Cells Using Homologous Recombination

Methods for modifying the genome of a Neural Stem Cell (NSC) are disclosed. Also, methods for differentiating NSCs into neurons and glia are described. NSCs are multipotent, self-renewing cells found in the central nervous system, capable of differentiating into neurons and glia. NSCs can be generated efficiently from pluripotent stem cells (PSCs) and have the capacity to differentiate into any neuronal or glial cell type of the central nervous system.

Methods of Treating or Preventing Pruritis (Itch)

This technology provides a novel method of treating or preventing pruritis (itch) using natriuretic polypeptide b (Nppb) blocking agents. Itch (also known as pruritis) is a sensation that may be perceived as an unpleasant skin irritation and may drive an urge to scratch. Conditions such as, for example, psoriasis, atopic dermatitis, renal failure, liver cirrhosis and some cancers may cause persistent itch. Itch is triggered by somatosensory neurons expressing the ion channel TRPV1 (transient receptor potential cation channel subfamily V member 1).

mNFHcre Transgenic Mice

Knockout mouse is a valuable model to study biological functions of target genes. When Cre expressing mice are bred with mice containing a loxP-flanked gene, the gene between the loxP sites will be deleted in the offsprings. Scientists at the NIH have generated mNF-H-cre transgenic mouse lines that express Cre recombinase under the control of the promoter of the neurofilament-H gene, which is expressed in the late stage of neuronal maturation. The transgenic mice express cre in neurons (but not astrocytes) with highest expression in the cortex and hippocampus.

3D Bioprinting of Cardiac Patch with Anisotropic and Perfusable Architecture for the Repair of Damaged Cardiac Muscle

This technology includes a novel cardiac patch which was 3D printed to repair damaged cardiac tissue. Based on biological and anatomical understanding of myocardial tissue, a novel 3D bioprinting technique was developed to directly fabricate the cellularized and vascularized cardiac patch with anisotropic fiber and perfusable vessel architecture. The design will integrate biomimetic aligned myocardial fibers and perfusable blood vessels to create a thick, functional cardiac patch, suitable for the human heart implantation.

Monoclonal Antibodies That Bind to the Underside of Influenza Viral Neuraminidase

Current influenza vaccines mainly induce antibodies against the surface glycoprotein hemagglutinin (HA) that block viral attachment to its host receptors and viral membrane fusion to the host cell. The immunodominant head region of HA undergoes antigenic drift and antibodies directed to the head confer little cross-protections between strains or subtypes.

Neutralizing Antibodies to Influenza HA and Their Use and Identification

The effectiveness of current influenza vaccines varies by strain and season, in part because influenza viruses continuously evolve to evade human immune responses. While the majority of seasonal influenza infections cause relatively mild symptoms, each year influenza virus infections result in over 500,000 hospitalizations in the United States and Europe. Current standard of care for individuals hospitalized with uncomplicated influenza infection is administration of neuraminidase inhibitors.

Programmable and Modular Nucleic Acid Nanoassemblies-based (NAN) Platforms to Regulate Mechanosensitive Activation of T-cells

This technology includes mechanobiological nucleic acid nanoassemblies-based platforms with dynamically controlled efficiency of T-cell activation. T-cells are the central players in adaptive immune response led by a T-cell receptor (TCR) centric machinery. Current T-cell activation strategy (e.g., micron-scale beads) focuses on 2D TCR-agonist biomimetic surfaces and biomimetic 2D immune synapses with planar traction, which requires non-physiological hyper-stimulatory cytokines levels (e.g., IL-2), and thus, is incompatible with clinical applications.

Henipavirus Vaccine

Henipaviruses are RNA viruses containing two high consequence human pathogens: Nipah virus (NiV) and Hendra virus (HeV). Both NiV and HeV infection in humans can result in severe respiratory disease and/or severe neurological manifestations, with mortality rates as high as 80%. There are currently no FDA-approved vaccines or therapeutics, and both NiV and HeV are considered dangerous emerging human pathogens with pandemic potential.

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