This technology includes an alpha-galactosidase-A knockout mouse model that can be used to study Fabry disease, an X-linked lysosomal storage disorder. Alpha-galactosidase-A is a crucial enzyme responsible for the breakdown of glycolipids, particularly globotriaosylceramide (Gb3), within lysosomes. In Fabry disease, a rare and inherited lysosomal storage disorder, mutations in the GLA gene lead to deficient or non-functional alpha-galactosidase-A enzyme activity.

This technology includes a novel vaccine for forming autoantibodies against apoC-III, a plasma enzyme that inhibits lipolysis. The vaccine can possibly be used to treat patients with high triglycerides and are at risk for pancreatitis and cardiovascular disease. This disclosure describes an ApoC3 immunogen that includes an antigenicApoC3 peptide linked to a bacteriophage virus-like-particle (VLP) immunogenic carrier.

The development of an effective HIV vaccine has been an ongoing area of research. High variability in HIV-1 virus strains, however,  represents a major challenge.  Ideally, an effective candidate vaccine would provide protection against the majority of clades of HIV.  Two major hurdles to overcome are immunodominance and sequence diversity. Researchers at the National Cancer Institute (NCI) have developed a vaccine that overcomes these major hurdles by utilizing a strategy that identifies conserved regions of the virus and exploits them for use in a targeted therapy.

The development of an effective HIV vaccine has been ongoing. HIV sequence diversity and immunodominance are major obstacles in the design of an effective vaccine. Researchers at the National Cancer Institute (NCI) developed a novel vaccine strategy combining both DNA and mRNA vaccination to induce an effective immune response. This combination strategy could also be used to develop vaccines against cancer or other infectious diseases (ex. SARS-CoV-2). 

The development of an effective HIV vaccine has been an ongoing area of research. The high variability in HIV-1 virus strains has represented a major challenge in successful development.  Ideally, an effective candidate vaccine would provide protection against the majority of clades of HIV.  Two major hurdles to overcome are immunodominance and sequence diversity.  This vaccine utilizes a strategy for overcoming these two issues by identifying the conserved regions of the virus and exploiting them for use in a targeted therapy. 

Scientists at NIAID have developed two immortalized stable B cell lines from rhesus macaques that can have value as research tools for the discovery of neutralizing antibodies of simian origin against HIV and that may have value in the development of an HIV vaccine. These B cell lines encode human Bcl-6 and Bcl-xL proteins, which are major regulators of apoptosis. These B cell lines are derived from the lymph node of a rhesus macaque (RM) that was infected with SHIV.CH505.

Despite four decades of intensive research, a safe and effective HIV-1 vaccine remains elusive due to the extreme difficulty in eliciting broadly neutralizing antibodies (bNAbs), which recognize and block HIV-1 from entering healthy cells. Only rare natural HIV-1 envelopes (Envs) promote the activation and expansion of naive B cells expressing unmutated germline antibodies of various bNAb lineages, but they typically do so for a single lineage for the same neutralization site.

NIAID scientists have developed plasmids that allow for production of pseudoviruses expressing SARS-CoV-2 spike protein. As SARS-CoV-2 is a lethal airborne virus, it must be handled in high-containment Biosafety Level 3 (BSL-3) laboratories that require strict airflow, ventilation and decontamination procedures.

The emergence of the SARS-CoV-2 virus and its immune-escaping variants have led to global COVID-19 pandemic/endemic, underscoring the urgent need for effective vaccines with strong and durable immune responses.