Scientists at The Wertheim UF Scripps Institute for Biomedical Innovation & Technology have recently been awarded a series of state and federal grants totaling approximately $15.7 million over the next five years. These substantial funds are expected to fuel groundbreaking research across several pivotal areas of biomedical science, including neuroscience, immunology, virology, and oncology. The institute, known for its interdisciplinary approach and cutting-edge innovations, continues to build on its robust annual research budget, which surpassed $101 million last year, contributing significantly to the University of Florida’s historic $1.33 billion research spending milestone.
At the forefront of this latest research funding is a diverse array of projects aimed at deciphering complex biological systems and developing novel therapeutic strategies. Among these, one of the most compelling focuses lies in the exploration of neuronal dynamics within the brain. Dr. Ezgi Hacisuleyman, an assistant professor at the institute, has secured a $2.3 million grant from the National Institute of General Medical Sciences. Her work probes the intricate mechanisms neurons use to rapidly alter their structure and function in response to external stimuli. By employing state-of-the-art imaging and molecular labeling techniques, Hacisuleyman’s research delves deeply into the subcellular localization and regulation of RNA and protein synthesis processes that enable neuronal function and plasticity, a pursuit critical to understanding neuropathologies rooted in cellular miscommunication.
Understanding how neurons strategically transport and locally produce RNA molecules challenges existing paradigms in molecular neuroscience. RNAs, beyond their classical role in protein synthesis, participate actively in regulating cellular responses, especially in polarized cells like neurons, where signaling and metabolic needs vary dramatically between dendrites, soma, and axon terminals. Hacisuleyman’s focus on these RNA localization mechanisms could unravel novel pathways implicated in neurological disorders such as Alzheimer’s disease, certain cancers, and inherited genetic conditions, paving the way for targeted RNA-based therapeutics.
Another major thrust of the institute’s research portfolio centers on combating pediatric HIV infection, a global health challenge that remains acute despite advances in antiretroviral therapy. Dr. Mauricio Martins has obtained a grant exceeding $6 million from the National Institutes of Health to explore innovative gene therapy techniques aimed at protecting infants from HIV transmission. In regions where access to conventional antiretroviral drugs is limited, preventing mother-to-child transmission during breastfeeding remains a formidable barrier. Martins’s pioneering work utilizes adeno-associated virus vectors to deliver broadly neutralizing antibody genes that offer durable protection in newborn rhesus macaques against simian-HIV, a promising model for blocking early HIV infection.
Intriguingly, the research uncovered an immune tolerance mechanism that is critical to the success of this gene therapy. Administering these protective antibodies shortly after birth risks triggering an immune response that neutralizes the therapy’s effectiveness. Here, the Martins team’s findings demonstrate that in utero exposure to neutralizing antibodies can induce immune tolerance, effectively preventing detrimental immune activation in infants. This discovery not only enhances the prospect of extending the therapeutic window for HIV prevention but also holds implications for modulating immune responses in autoimmune diseases and improving outcomes in organ transplantation.
Addressing the pressing clinical challenge of cancer relapse, Drs. Michalina Janiszewska and Matthew Disney are leveraging a $300,000 state grant to develop new therapeutic approaches targeting glioblastoma, the most aggressive and lethal type of brain tumor in adults. Despite standard treatment modalities combining chemotherapy and radiation therapy, patient survival gains have remained marginal for over a decade. The team’s research focuses on the tumor’s adaptation to hypoxic conditions — low oxygen environments — which fosters tumor resilience and resistance to therapy.
Their strategy hinges on inhibiting hypoxia-inducible factors, specifically HIF2-alpha, a transcription factor that orchestrates cellular responses to oxygen deficiency and drives tumor progression. By integrating expertise in chemical biology and medicinal chemistry, the researchers are designing small molecules capable of selectively binding messenger RNAs (mRNAs) involved in hypoxia signaling. Targeting the mRNA of HIF2-alpha represents an innovative therapeutic angle that disrupts gene expression at the RNA level, offering precision in modulating pathological pathways. This approach holds promise not only for glioblastoma but potentially for breast cancer metastasis and other hypoxia-associated malignancies.
Further contributing to the antiviral armamentarium, Dr. Susana Valente’s laboratory has secured a five-year, $4.8 million grant from the National Institute of Allergy and Infectious Diseases to advance a novel class of HIV inhibitors. Rather than targeting viral entry or replication enzymes, Valente’s group focuses on Tat, an essential viral protein that functions as a trans-activator of HIV gene transcription. Tat activates the virus from a latent state, initiating a cascade of viral production and toxicity within infected cells. By triggering the cell’s ubiquitin-proteasome system to selectively degrade Tat, Valente’s team is developing molecules that ‘turn off’ the virus’s transcriptional switch, implementing a sophisticated block-and-lock tactic aimed at durable suppression of HIV without ongoing therapy.
This novel Tat-targeting strategy represents a paradigm shift in HIV therapeutics, potentially enabling treatment-free viral remission and reducing the burden of lifelong antiretroviral drug regimens. The research integrates medicinal chemistry innovations with rigorous in vitro and in vivo evaluations, including humanized mouse models and ex vivo human cell systems, to optimize drug candidates and verify their efficacy and safety profiles. Success in this endeavor could revolutionize the management of chronic viral infections and inspire similar transcription-targeted antiviral approaches.
Collectively, these research initiatives exemplify The Wertheim UF Scripps Institute’s commitment to advancing biomedical science through multidisciplinary collaboration and translational research. By harnessing expertise in molecular biology, chemistry, immunology, and clinical science, the institute seeks to translate fundamental discoveries into innovative treatments that address some of the most challenging diseases facing humanity today.
Founded in partnership with Scripps Research and integrated into the University of Florida’s ecosystem in 2022, the institute benefits from a dynamic environment fostering synergy between basic research and drug discovery. This recent infusion of funding underscores confidence in the institute’s vision and capability to impact global health positively.
The awarded grants not only bolster ongoing projects but also cement the institute’s role as a hub for scientific breakthroughs ranging from decoding neuronal communication to developing next-generation antiviral and anticancer therapies. As these research programs unfold, the biomedical community anticipates new insights and clinical applications that may transform treatment paradigms and improve patient outcomes worldwide.
With its distinct blend of foundational science and innovative translational approaches, The Wertheim UF Scripps Institute stands as a model for how interdisciplinary collaboration and sustained investment in biomedical research can accelerate progress toward curing diseases that have long eluded effective therapies.
Subject of Research: Biomedical innovation focusing on neuroscience, HIV treatments, cancer therapeutics, and RNA-targeted drug development.
Article Title: Scientists at The Wertheim UF Scripps Institute Awarded $15.7 Million to Advance Next-Generation Biomedical Research
News Publication Date: Not specified
Web References:
https://directory.ufhealth.org/griffin-patrick-1
https://directory.ufhealth.org/hacisuleyman-ezgi
https://directory.ufhealth.org/de-aguiar-martins-mauricio
Gene Therapy May Block HIV Transmission During Breastfeeding, Study Shows
https://directory.ufhealth.org/janiszewska-michalina
https://directory.ufhealth.org/disney-matthew
https://directory.ufhealth.org/valente-susana
Image Credits: The Wertheim UF Scripps Institute
Keywords: Molecular neuroscience, HIV treatments, biomedical innovation, HIV gene therapy, Tat inhibitor, glioblastoma, hypoxia-inducible factors, RNA therapeutics, antiviral drug development, immune tolerance, pediatric HIV, cancer relapse prevention
Tags: biomedical innovation fundingDr. Ezgi Hacisuleyman research grantgroundbreaking therapeutic strategiesimmunology and virology studiesinterdisciplinary biomedical scienceneuronal dynamics explorationneuroscience research advancementsoncology research projectsRNA and protein synthesis mechanismsstate and federal research fundingUniversity of Florida research budgetWertheim UF Scripps Institute research grants
