Oral Treatment of Hemophilia

This invention portrays a simple method for treatment of antigen-deficiency diseases by orally administering to a subject a therapeutically effective amount of the deficient antigen, wherein the antigen is not present in a liposome. This method increases hemostasis in a subject having hemophilia A or B, by orally administering to the hemophiliac a therapeutically effective amount of the appropriate clotting factor, sufficient to induce oral tolerance and supply exogenous clotting factor to the subject.

Stable Cell Line Technology for Enhanced Production of Varicella-Zoster Virus Vaccines

This technology includes a stable cell line engineered to have reduced expression of the pro-apoptotic protein Bim when exposed to doxycycline. This reduction in Bim expression allows for significantly higher yields of the Varicella-Zoster Virus (VZV), which is used in the production of live, attenuated VZV vaccines. The enhanced viral production makes this cell line particularly useful for vaccine manufacturing.

Derivation of a >25 million-year-old Adeno-associated Virus Coat Protein Sequence for Gene Transfer Studies

This technology includes a novel capsid protein for recombinant adeno-associated virus (AAV)-mediated gene transfer evaluation. We have identified a "fossilized" endogenous AAV sequence element (referred to as mAAV-EVE) within the germline of an ancient lineage of Australian marsupials and have cloned and sequenced mAAV-EVE orthologs from at least fifteen lineage-specific taxa.

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.
Subscribe to Pre-Clinical (in vitro)