Main Menu

Currently available identification methods for antigen-specific antibodies require live pathogens, antisera (that are only available for a limited number of species), and species-specific secondary antibodies (also a limited resource). Thus, detection or surveillance of pathogens in wild avian species or zoo animals, for example, is complex and cumbersome.

Investigators at the National Heart, Lung, and Blood Institute have designed a novel lentiviral vector as a potential gene therapy for sickle cell anemia and beta-thalassemia. The novel lentiviral vector encodes the beta-globin gene in a forward orientation and can produce 5-10 fold higher viral titer and 4-10 fold higher gene transfer efficiency to hematopoietic stem cells than reverse-oriented lentiviral vectors. In vivo studies conducted in rhesus macaques show beta-globin production after transplantation with this novel lentiviral vector.

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 core proteins of HIV-1 are secreted into the environment during replication in the human body. The detection of the core protein p24 (molecular mass of 24 kilodaltons) serves as an indicator of early HIV-1 infection, and assays detecting it have been available since the late 1980s. However, the development of a rapid assay for the detection of HIV-1 p24 has only recently become available.

Upon intranasal vaccination, live attenuated influenza vaccine (LAIV) viruses may replicate within the nose for several days. Current clinical diagnostic tests cannot distinguish between LAIV viruses and multiple influenza viruses in recently inoculated patients that present with respiratory symptoms. This poses a problem for the diagnosis and treatment of patients with respiratory symptoms, as these symptoms may not be caused by influenza. CDC researchers have developed a real-time RT-PCR assay to detect the presence of LAIV viruses.

Available for licensing and commercial development as imaging agents for positron emission tomography of cancer are boramino acid compounds. The inventors showed that mimetics created by substituting the carboxylate group (-COO^-) of an amino acid with trifluoroborate (-BF_3^-) are metabolically stable and allow for the use of fluorene-18 (¹⁸F) as the radiolabel.

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.

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.

Herpes simplex viral infections, including herpes simplex virus type 1 (HSV-1) and type 2 (HSV-2), are exceptionally common worldwide. These viruses establish lifelong persistent infections with cycles of lytic reactivation to produce recurrent diseases including oral and genital lesions, herpetic keratitis/blindness, congenital-developmental syndromes, and viral encephalitis. Infection with HSV-2 increases the rate of human immunodeficiency virus (HIV) transmission in coinfected individuals. DNA replication inhibitors are typically used to treat herpesvirus infections.