Main Menu

Adeno-associated viruses (AAV) are used in gene delivery, but with limited success due to toxicity. The novel AAVs described in this technology may be more effective and useful in gene therapy applications.

The invention provides a method of diagnosing X-linked severe combined immunodeficiency (XSCID) in males or determining whether females are carriers. This method is based upon the presence of either a mutated or truncated IL-2Rgamma gene. The invention also discloses a method of treating XSCID as well as a method for monitoring the therapy.

Transformation-Associated Recombination (TAR) cloning in yeast is a unique method for selective isolation of large chromosomal fragments or entire genes from complex genomes without the time-consuming step of library construction.¹ The technique involves homologous recombination during yeast spheroplast transformation between genomic DNA and a TAR vector that has short (approximately 60bp) 5’ and 3’ gene targeting sequences (hooks).

Available for licensing is the A.E7 T cell clone, a model Th1 clone described in Matis et al., J Immunol. 1983 Apr;130(4):1527-1535 [PubMed abs] and J Immunol. 1983 Sep;131(3):1049-1055 [PubMed abs].

Ixodes scapularis is a blood-sucking tick and the principal vector of Lyme disease, a spirochetal illness caused by Borrelia burgdorferi and now the most common vector-borne infection in the United States; more than 50,000 cases have been reported during the last ten years. The salivary gland of I. scapularis has a number of pharmacologically active molecules that help the tick to successfully feed on blood, such as inhibitors of complement system, in addition to coagulation and platelet aggregation inhibitors.

One of the major limitations of using cultured keratinocytes for research studies is that primary keratinocytes senesce after a few passages. Keratinocytes from specific anatomical sites are also difficult to culture. Scientists at the NIH have demonstrated that primary keratinocytes, from several anatomical sites, when treated with a small-molecule inhibitor of the ROCK protein maintain a proliferative state and become immortal without genetic modification to the cells.

Malaria is the single leading cause of death, especially among children, in the developing world. Malaria is caused by infection with parasites of the genus Plasmodium, transmitted by mosquitos. In addition to transmission, vital steps in the parasite lifecycle occur in the mosquito host. The invention offered for licensing relates to therapeutic compounds and related pharmaceutical compositions that can be used in the prevention and treatment of malaria infection.

Monitoring an immune system that has been depleted by infection (e.g., HIV), chemotherapy, or progenitor cell transplantation is vital to assessing individual’s recovery status. This technology provides a new plasmid standard to be used as part of the existing TREC assay. This new plasmid has a shorter insert than the commercially available one, which means it now matches the PCR product generated in the qPCR reaction in the TREC assay. Additionally, the new plasmid is easier to grow up than the existing standard.

Available for licensing are methods to generate T cells responsive to multiple polyomaviruses. The resulting T cell populations could be useful in treating immunosuppressed individuals with polyomavirus infections or polyomavirus-associated pathologies such as Merkel cell carcinoma (MCC), polyomavirus-associated nephropathy (PVAN), hemorrhagic cystitis, progressive multifocal leukoencephalopathy (PML), and trichodysplasia spinulosa (TS). The methods could also be used to restore polyomavirus-specific immunity in immunocompromised individuals.

The Molecular Recognition Section of NIDDK announces the availability of a novel triazole-based probes, structures which act as antagonists at human P2Y₁₄ receptors. Although the physiologic functions of this receptor remain undefined, recently it has been strongly implicated in immune and inflammatory responses. Prior work with a 4,7-disubstituted 2 naphthoic acid derivative (PPTN) established the ability to inhibit chemotaxis of human neutrophils in the lung and kidney.