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Patient conditioning is a critical initial step in hematopoietic stem and progenitor cell (HSPC) transplantation procedures to enable marrow engraftment of infused cells. Conditioning regimens have traditionally been achieved by delivering cytotoxic doses of chemotherapeutic agents and radiation. However, these regimens are associated with significant morbidity and mortality, and cannot be used safely in elderly or subjects with comorbidities.

This technology includes an alternative source of plasmid DNA produced in eukaryotic cells for non-viral gene transfer, which represent a novel eukaryotic alternative to bacterial plasmid DNA. Once introduced into non-dividing cells, ceDNA persists and transgene expression remains stable whereas plasmid (p) DNA is lost. The ceDNA and transfection reagent complex is nonimmunogenic allowing re-administration as needed: recombinant adeno-associated virus (rMV) is immunogenic precluding repeated administration.

This technology includes a strategy to target tumor cells that lost antigen following reaction with a therapeutic antibody by targeting the complement component C3d that has been deposited on target cells by the primary antibody. We previously generated a C3d-specific mouse/human chimeric antibody called C8xi and obtained proof of principle for the approach in two preclinical models. Here we summarize the generation of a new set of C3d targeting antibodies.

The invention provides identification methods for agents which inhibit the Jak-STAT signaling transduction pathway. Drugs identified by these methods are candidates for the treatment of proliferative disorders dependent on the Jak-STAT pathway, including those caused by HTLV-1. In addition, such agents may be potent immunosuppressive drugs with potential applications not only for organ transplantation but also for treatment of autoimmune diseases.

The invention described and claimed in these patent applications provides for novel hybrid vectors which may be used for cell transformation either in vivo, in vitro, or ex vivo. The hybrid vectors, which are capable of integrating into the chromosome of the host cell and are capable of transducing dividing and non-dividing cells, have an adenoviral serotype 5 backbone and two retroviral (Moloney murine leukemia virus) elements upstream and downstream of the transgene.

Hearing loss is a common communication disorder affecting nearly 1 in 1,000 children in the United States alone, and nearly 50% of adults by the age of eighty. Hearing loss can be caused by environmental and disease-related factors; however, hearing loss due to genetic factors accounts for approximately 50% of cases.

This invention claims antibodies and/or fragments thereof specific for hepatitis A virus (HAV) and the use of the antibodies in the diagnosis, prevention, and treatment of hepatitis A. Hepatitis A is the most common type of hepatitis reported in the United States, which reports an estimated 134,000 cases annually, and infects at least 1.4 million people worldwide each year. HAV is a positive sense RNA virus that is transmitted via the fecal-oral route, mainly through contaminated water supplies and food sources.

Leishmania parasites are transmitted to their vertebrate hosts by infected phlebotomine sand fly bites. Sand fly saliva is known to enhance Leishmania infection, while immunity to the saliva protects against infection. This invention claims nine major salivary proteins from the sand fly vector of Leishmania major, Phlebotomus papatasi, nucleic acids encoding the proteins, vaccines comprising the proteins and/or nucleic acids, and methods of producing an immune response to prevent Leshmaniasis.

Adeno-associated virus (AAV) is being developed for gene therapy applications. This virus type presents several advantages over alternate vectors for therapeutic gene delivery. AAV is not considered pathogenic and transduces stably dividing and non-dividing cells. AAV also shows good serotype specificity to various cell types for targeted gene delivery.

West Nile virus (WNV) has recently emerged in the U.S. and is considered a significant emerging disease that has embedded itself over a considerable region of the U.S. WNV infections have been recorded in humans as well as in different animals. From 1999-2014, WNV killed 1,765 people in the U.S. and caused severe disease in more than 41,762 others. This project is part of NIAID's comprehensive emerging infectious disease program.