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“Evaluation of the nasal probiotic candidate Dolosigranulum pigrum in an infant non-human primate model.” Respiratory tract infections are the leading infectious cause of death among children. Increasingly, the bacteria that inhabit the human upper respiratory tract – referred to as the microbiome – are recognized to influence the risk and severity of respiratory tract infections during children. In particular, these studies have demonstrated that an understudied bacterium, Dolosigranulum pigrum, is important for the maintenance of respiratory health. In this project, we will determine the safety and optimal dosing of this bacterium when administered as a nasal probiotic to infant rhesus macaques, providing data that will support future experiments to evaluate its effectiveness for respiratory infection prevention.

Dr. Matthew Kelly, M.D., M.P.H,Duke University School of Medicine, Durham, NC

“Discovering ocular biomarkers for the pathogenesis of Alzheimer’s disease in a non-human primate model.” This study explores how changes in the retina may signal Alzheimer’s Disease (AD) in nonhuman primates. By comparing retinal images and brain tissue from older primates, we hope to find early warning signs of AD that are easy to detect by non-invasive eye imaging. If successful, this could lead to new ways for early accurate diagnosis of AD and ultimately help to develop measurements for treatment response monitoring for the disease.

Dr. Allison Liu, M.D., Ph.D.,UC Davis School of Medicine, Davis, CA

“The role of posttranscriptional gene regulation during neocortical specification.” Neural progenitors in the neocortex give rise to the different classes of projection neurons and glia in a conserved order, but the molecular mechanism(s) coordinating cell fate determination with developmental time remains unknown. We hypothesize that a pathway involving miRNAs is responsible for controlling the changes in cortical progenitor competence and further propose a broad role for posttranscriptional regulation in cortical cell fate specification.

Dr. Sergi Simó, Ph.D.,UC Davis School of Medicine, Davis, CA

“Advancing gene therapy for oculocutaneous albinism.” Oculocutaneous albinism (OCA) is a group of disorders that causes reduced pigmentation in the eyes, hair, and skin, resulting in severe visual impairment. Currently, there are no effective treatments for OCA vision loss, making it a significant unmet clinical need. Gene therapy is a promising approach for treating OCA, but we lack animal models with eyes similar to humans in which to develop and test these therapies. Recently, several adult rhesus macaques at CNPRC were identified as having features similar to humans with OCA, including reduced pigmentation in the hair and eye. In this proposal, we will screen NHPs at CNPRC to identify carriers of OCA-causing mutations. We will also perform preliminary testing of OCA gene therapy in primates. Overall, this research may lead to the development of a potential gene therapy treatment for OCA and improve the quality of life for those affected by this disorder.

Dr. Leah Byrne, Ph.D.,University of Pittsburgh School of Medicine, Pittsburgh, PA

“Novel strategy to enable re-administration of vectors for gene therapies and vaccines.” Gene therapy with adeno-associated virus (AAV) vector has been successfully applied in numerous clinical trials with 6 AAV based drugs approved by the FDA recently. However, more than 50% of patients have antibodies against AAV and been excluded from this effective treatment. We have developed a biomolecule with high efficiency to block AAV antibodies in mice. In this study, we will study the efficacy and safety of this biomolecule in non-human primates. If successful, this biomolecule could be immediately transited into the clinical applications to benefit more patients receiving AAV gene therapy.

Dr. Chengwen Li, M.D., Ph.D.,University of North Carolina School of Medicine, Chapel Hill, NC

“Immunomodulatory effects of clostridium immunis in a non-human primate model.” Numerous human diseases are thought to result from an abnormal immune response against the bacteria that normally live in and on every individual (the microbiota), with the idea that “fixing” this microbial community may treat the disease itself. We recently discovered a new human bacterium, Clostridium immunis, that protects mice against metabolic and inflammatory diseases by regulating the immune system. In this pilot project, we aim to determine the safety and efficacy of C. immunis in rhesus macaques. These studies will provide pivotal pre-clinical findings that will help develop C. immunis into a clinically-relevant therapeutic.

Dr. Neeraj Surana, M.D., Ph.D.,Duke University, Durham, NC

“Evaluating the safety and efficacy of CD47-IL6 blockage by antibody for reversing pulmonary fibrosis in interstitial lung diseases in a non-human primate model.” Pulmonary fibrosis (PF) and architectural remodeling of tissues disrupt lung function with often fatal consequences. Currently, there are no successful treatments for reversing lung fibrosis. Our previous research revealed effective anti-fibrotic therapeutic strategies to obtain reversion of interstitial lung disease in long COVID patients with PF. Our anti-fibrotic therapeutic strategy is to resolve lung fibrosis in our pre-clinical models at the transcriptional level with significant therapeutic efficacy and minimal or no toxicity. Our goal is to further our approach in non-human primate models for further evaluation of the toxicity and efficacy of our studies.

Dr. Gerlinde Wernig, M.D.,Stanford University, Stanford, CA