Jakob Körbelin is a Principal Investigator at the University Medical Center Hamburg-Eppendorf (UKE), affiliated with the Faculty of Medicine and the II. Medical Clinic and Polyclinic. His research focuses on vascular biology, gene therapy, and neurological disorders, particularly targeting the blood-brain barrier using adeno-associated viral (AAV) vectors. He leads studies on pulmonary hypertension, neurovascular interactions, and genetic diseases like Niemann-Pick type C2. His work bridges basic science and translational medicine, emphasizing AAV engineering and therapeutic applications. Key research interests include endothelial cell biology, neuroinflammation, and the pathophysiology of vascular diseases. Notably, he received the UCCH Hubertus Wald Young Investigator Award 2013 for his contributions. His lab explores mechanisms of vascular dysfunction, gene delivery optimization, and the impact of viral vectors in treating rare diseases. Collaborations span molecular neurobiology, immunology, and translational oncology. Publications highlight breakthroughs in AAV-mediated therapies, such as reversing neurodegeneration in NPC2 models and identifying novel targets for pulmonary hypertension. Ongoing projects address microvascular brain pathology in SARS-CoV-2 infection and the role of transcription factors in vascular diseases.
Michael Daniele is an Associate Professor at North Carolina State University, jointly appointed in the Department of Electrical & Computer Engineering and the Joint Department of Biomedical Engineering . His research focuses on bioelectronics engineering, particularly in developing microsystems for monitoring, mimicking, and augmenting biological functions. He leads the @BiointerfaceLab , exploring wearable/implantable biosensors, microphysiological systems, and process analytical technologies for biomanufacturing. Education : Ph.D. in Materials Science & Engineering (Clemson University, 2012) Bachelor's in Materials Science & Engineering (Rutgers University, 2009) Research Highlights : Developing "injury-on-a-chip" models for coagulation studies Pioneering hydrogel microneedles for diagnostic devices Advancing light-controlled peptide ligands for protein purification Collaborating with Novartis on viral vector manufacturing Award Recognition : 2024 William F. Lane Outstanding Teaching Award 2019 NSF CAREER Award 2022 University Faculty Scholar Grants & Initiatives : Co-leader of the NC-Viral Vector Initiative (2023–present) NSF-funded projects in biosensor integration and biomanufacturing His work bridges engineering and medicine, with applications in gene therapy, wearable diagnostics, and precision agriculture.
Dr. Athma A Pai is an Associate Professor at UMass Chan Medical School, holding primary appointments in the RNA Therapeutics Institute and the T.H. Chan School of Medicine. She maintains extensive secondary appointments across multiple departments including Genomics and Computational Biology, Systems Biology, and several graduate programs at the Morningside Graduate School of Biomedical Sciences, reflecting the highly interdisciplinary nature of her work. Education: BS in Biochemistry/Anthropology from University of Pennsylvania PhD in Human Genetics from University of Chicago Postdoctoral training in RNA Genomics from MIT Dr. Pai's research program centers on RNA biology with particular emphasis on RNA processing, splicing mechanisms, and the regulation of gene expression. Her work investigates how environmental factors influence RNA processing through biochemical, molecular, and genetic mechanisms. She employs cutting-edge genomic and transcriptomic approaches to study alternative polyadenylation, mRNA transcript initiation and termination, and the spatial organization of RNA processing events within cells. Her research has significant implications for understanding fundamental gene regulation mechanisms and their roles in disease processes. Analysis of Dr. Pai's recent publications reveals a strong focus on developing high-resolution profiling methods for understanding transcriptional and translational regulation. Her work increasingly integrates computational approaches with experimental biology to investigate how RNA processing events are coordinated across the transcriptome. A notable trend is her exploration of how RNA processing contributes to inflammatory responses and cellular defense mechanisms, with implications for therapeutic development. Dr. Pai actively mentors students through multiple graduate programs at UMass Chan Medical School, including Biochemistry and Molecular Biotechnology, Biophysical Chemical and Computational Biology, Interdisciplinary Graduate Program, MD/PhD Program, RNA Therapeutics and Biology Program, and Systems Computational and Quantitative Biology. She maintains an active laboratory (Pai Lab) that welcomes postdoctoral researchers interested in RNA biology. Her laboratory website provides additional information about ongoing research projects and opportunities for collaboration and training, and she maintains a professional presence through her Twitter account (@athmapai).
Marc Sommer is a Professor of Biomedical Engineering and Psychology & Neuroscience at Duke University. He directs the Duke Institute for Brain Sciences and contributes to the Duke Initiative for Science & Society. His research focuses on neural circuits for cognition , particularly how the brain maintains visual perception and behavior through interactions between frontal cortex and subcortical regions. Key Techniques : Single neuron electrophysiology, optogenetics, psychophysics, computational modeling Translational Goals : Improving transcranial magnetic stimulation (TMS) and viral vector therapies for psychiatric and motor disorders Scientific Awards : Capers and Marion McDonald Award for Excellence in Teaching and Research (2021) Capers and Marion McDonald Award for Excellence in Mentoring and Advising (2017) Bass Fellow, Duke University (2017) Research Fellowship in Neuroscience, Alfred P. Sloan Foundation (2005) Marc Sommer's recent publications span visual stability across saccades , optogenetics in primates , TMS mechanisms , and viral vector applications . His work bridges cognitive neuroscience and biomedical innovation , with significant contributions to corollary discharge theory and neural circuit mapping . Lab Collaborations : Interdisciplinary partnerships across Duke University and external institutions, integrating engineering , neurobiology , and computer science to decode primate brain circuitry.
William A. Nyberg is an Assistant Professor at the Department of Medicine, Huddinge, Karolinska Institutet. His research focuses on the in vivo genetic modification of T cells for cancer treatment, utilizing chimeric antigen receptors (CARs), CRISPR/Cas9, and synthetic vectors like adeno-associated viruses (AAVs) and lipid nanoparticles (LNPs). The lab aims to eliminate time-consuming ex vivo manufacturing processes and enhance CAR-T cell efficacy in immunosuppressive tumor microenvironments. Assistant Professor, Department of Medicine, Huddinge (2024–2030) SciLifeLab Fellow (2024–2030) European Research Council grant recipient (2025–2029) Recruiting postdoctoral fellows, doctoral, and master's students His work intersects Genetic Engineering, Immunology, and Oncology, with key subfields including CAR-T cell therapy, CRISPR/Cas9, tumor microenvironment, and synthetic biology. Collaborative projects involve Yenan Bryceson's group and translational humanized/syngeneic mouse models. Scientific Awards: SciLifeLab Fellow (Karolinska Institute, 2024–2030) Advising: Julian Fischbach (Doctoral student)
Masmudur Rahman is an Assistant Professor at Arizona State University (ASU), affiliated with the School of Life Sciences and the Biodesign Center for Personalized Diagnostics. He leads research on molecular virology, focusing on oncolytic viruses like myxoma virus (MYXV) to treat cancers, particularly leveraging their ability to target cancer cells with compromised innate immunity. His work combines virology, genetic engineering, microscopy, and bioinformatics. Education highlights include a BSc and MSc from the University of Dhaka, a PhD in Molecular Virology from the Indian Institute of Science (IISc), and postdoctoral research at the University of Western Ontario and University of Florida, studying poxvirus immune modulation. He has received grants from NIH and the Arizona Biomedical Research Commission for projects on cancer virotherapy and virus-host interactions. Research interests span viral host range mechanisms, tumor tropism, and combination therapies (e.g., selinexor + MYXV). His lab develops strategies to enhance viral efficacy using stem cells and explores autosis induction in CAR-T cells. He teaches undergraduate research and honors courses in microbiology and cell biology, emphasizing hands-on training in virology techniques. Grants include NIH-funded studies on overcoming CAR-T resistance via autosis and targeting metastatic cancers with MYXV-selinexor combinations. His work has been published in Viruses , Cancer Research Communications , and International Journal of Molecular Sciences , among others.
Scot Wolfe is a Professor at UMass Chan Medical School, affiliated with the Department of Molecular, Cell and Cancer Biology. He holds multiple academic roles across institutions including T.H. Chan School of Medicine and Morningside Graduate School of Biomedical Sciences. His research focuses on engineering CRISPR-Cas9 systems for gene therapy, targeting diseases like sickle cell anemia and muscular dystrophy. Wolfe's work emphasizes precision genome editing, delivery systems, and applications in hematopoietic stem cell therapy. Collaborations include projects on HIV latency, zebrafish genomics, and Neurofibromin gene therapy. His lab (wolfe-lab.org) innovates tools like Cas9-ZFP fusions and delivery methods for clinical applications. Education: B.S. from Caltech (1990), Ph.D. from Harvard (1996). Postdoc at MIT. Research spans protein-DNA recognition, zinc finger engineering, and transcription factor specificity. Over 150+ publications focus on gene editing mechanisms and therapeutic applications. Active in the NIH Somatic Cell Genome Editing program. Key projects include enhancing CRISPR specificity, ex vivo editing of HSPCs, and combating genetic disorders via base editing and prime editing. His team develops therapies for beta-hemoglobinopathies, limb-girdle muscular dystrophy, and Hermansky-Pudlak Syndrome. Ongoing work explores HIV cure strategies through proviral inactivation.
Lars Aagaard is an Associate Professor at the Department of Biomedicine - Research and Education, Aarhus University, Denmark. He is based at the Bartholin Building in Aarhus C and is actively engaged in research and teaching in the fields of gene therapy, RNA interference, and genome editing. His work focuses on developing viral vectors, particularly AAV and lentiviral vectors, for ocular gene therapy targeting retinal diseases such as age-related macular degeneration and diabetic retinopathy. His research interests include the development of Dicer-independent RNAi systems, combinatorial gene therapy using microRNA and CRISPR/Cas9, and the delivery of RNA- and protein-based therapeutics. He also teaches Genetics and Personalized Medicine and supervises postdoctoral researchers, PhD, master's, and undergraduate students. The recent publications (2022–2024) reflect a strong trend in RNAi therapeutics, viral vector engineering, and translational applications in ocular and systemic diseases. Themes include gene silencing, immune modulation, and regenerative approaches using engineered cells. Aagaard actively contributes to the scientific community as a reviewer for journals such as Nature and Molecular Therapy . He has participated in key international conferences including ARVO, ASGCT, and ESGCT, demonstrating ongoing engagement with the global gene therapy community. Supervision of postdocs, PhD, master’s, and undergraduate students Active participation in international conferences and workshops Collaborative research across institutions, including Universidade Federal de Sao Paulo He is involved in both basic and translational research, with potential for clinical applications in gene and cell therapy.
Edward F. Chang, MD is a distinguished Professor and Chair of the Department of Neurological Surgery at the University of California, San Francisco (UCSF) School of Medicine. He co-directs the Center for Neural Engineering and Prostheses, a collaborative enterprise between UCSF and UC Berkeley, and leads the Chang Lab focused on speech neuroscience and neural engineering. As a practicing neurosurgeon, he specializes in treating adults with difficult-to-control epilepsy, brain tumors, trigeminal neuralgia, hemifacial spasm, and movement disorders. Dr. Chang's educational background includes a B.A. in Chemistry from Amherst College (1997), an M.D. from UCSF (2004), a Neurological Surgery residency at UCSF (2010), and a postdoctoral fellowship in Cognitive Neuroscience at UC Berkeley (2009). His research focuses on the brain mechanisms for speech, movement, and learning, with particular emphasis on advanced brain mapping methods to preserve crucial areas for speech and motor functions. He has pioneered work in speech neuroprostheses, developing technology that allows patients with paralysis to communicate through brain signals. His work integrates engineering, neurology, and neurosurgery to develop state-of-the-art biomedical technology to restore function for patients with neurological disabilities such as paralysis and speech disorders. Analysis of his recent publications reveals a strong trend toward developing advanced neuroprosthetic technologies, particularly speech decoding systems, and exploring the neural basis of speech production across multiple languages. His research also spans epilepsy surgery optimization, deep brain stimulation for psychiatric conditions, and molecular profiling of brain tumors. Blavatnik National Laureate for Life Sciences (2015) Elected to the National Academy of Medicine (2020) Inaugural Bowes Biomedical Investigator at UCSF HHMI Faculty Scholar Dr. Chang leads multiple NIH-funded research projects totaling millions of dollars, including a pilot clinical trial for speech neuroprosthesis and studies on the neural coding of speech across human languages. He has mentored numerous researchers in the field of neural engineering and speech neuroscience, though specific student names aren't listed in the provided materials. His work has resulted in groundbreaking technologies that have helped restore communication abilities to individuals with paralysis. As co-director of the Center for Neural Engineering and Prostheses, Dr. Chang leads a multidisciplinary team of engineers, neurologists, and neurosurgeons working at the intersection of neuroscience and technology. His lab has been instrumental in developing brain-computer interfaces that translate neural activity into speech, with recent publications demonstrating streaming brain-to-voice neuroprostheses that restore naturalistic communication.
Simon Liang serves as an Assistant Professor in the Department of Medicine at the University of Minnesota School of Medicine, specializing within the Gastroenterology, Hepatology, and Nutrition Division. His research integrates genome editing technologies with cancer therapeutics, focusing on ADAR1-mediated RNA editing pathways in hematologic and solid tumors. Dr. Liang's research interests center on developing CRISPR-based therapeutic strategies for cancer treatment, with particular emphasis on ADAR1 inhibition as a novel approach for acute myeloid leukemia and prostate cancer. His work bridges genome editing , epigenetic regulation , and RNA biology to address therapeutic resistance mechanisms. Key technical expertise includes prime editing optimization, CRISPR delivery systems (particularly AAV vectors), and methylation analysis. Analysis of his publication trends reveals a strategic evolution from foundational CRISPR methodology development (2019-2021) toward therapeutic applications in oncology (2022-2025), with increasing focus on translational models including non-human primates. His most impactful recent work demonstrates ADAR1's critical role in cancer cell survival across multiple malignancies. While specific awards aren't documented in the profile, his research has garnered significant attention with PlumX metrics showing: Multiple publications picked up by news outlets Substantial social media engagement (X, Bluesky) High Mendeley reader counts indicating scholarly impact Scopus citations accumulating rapidly for 2024-2025 publications Dr. Liang actively collaborates across disciplines as evidenced by co-authorship with experts in virology, oncology, and gene therapy. His work contributes to UN Sustainable Development Goals related to good health and well-being through development of precision cancer therapeutics. Current research directions include advancing prime editing for clinical translation and exploring ADAR1 inhibitors in combination therapies.
Lee Hyuk-jin is a Professor at the Department of Biomedical Engineering, College of Engineering, Seoul National University. He holds a Ph.D. in Life Sciences from KAIST (2009) and completed prior degrees at Columbia University (M.S. in BME, 2004) and Johns Hopkins University (B.A. in BME, 2002). Ph.D., Life Sciences, KAIST (2009) M.S., Biomedical Engineering, Columbia University (2004) B.A., Biomedical Engineering, Johns Hopkins University (2002) His research focuses on RNA-based gene/cell therapy and RNA nanotechnology , with a specialization in lipid nanoparticle (LNP) engineering. Recent work includes in vivo RNA delivery systems for hemophilia A, pulmonary fibrosis, and innate immune responses to mRNA vaccines. Notable trends in his publications include: advances in LNP design for targeted RNA delivery, therapeutic applications in liver and lung diseases, and immune modulation mechanisms for vaccines. Awards include the 2023 Science and Technology Medal on Science Day. Recipient of 220 million KRW research grant (5 years) from the Ministry of Science and ICT Led development of innovative biomaterials for gene therapy
Dr. Harsh Vardhan is a Postdoctoral Research Associate and Rice Academy Junior Fellow at Rice University's Department of Chemical & Biomolecular Engineering, working under Dr. Rafael Verduzco. His research focuses on covalent organic frameworks (COFs) for applications in PFAS degradation and selective metal ion removal from water. Previously, he held roles as a postdoctoral fellow at Northwestern-Argonne Institute of Science and Engineering and conducted doctoral research on COFs for heterogeneous catalysis at the University of South Florida. He has been recognized with multiple awards, including the Alexiou Award in Environmental Chemistry and Rice Academy Fellowship. Education: Ph.D. in Chemistry (University of South Florida, 2021), M.S. in Chemistry (Indian Institute of Technology-Kharagpur). His work bridges materials science, environmental engineering, and nanotechnology. Key projects include developing COF-based materials for ion rejection and catalytic processes, alongside pioneering CRISPR-Cas9 genome editing tools for therapeutic applications in sickle cell disease. Research Interests: Catalytic materials design Environmental remediation technologies Nanomaterials synthesis CRISPR-based gene editing Water purification systems Publications Trends: Over 15 peer-reviewed articles highlight his contributions to genome editing precision, CRISPR off-target mitigation, and COF material applications. Recent work emphasizes spatial control of gene editing and AAV vector engineering. Awards & Grants: Recipient of departmental research grants, travel awards, and student-nominated teaching accolades. Current funding includes the Rice Academy Fellowship supporting his PFAS degradation studies. Labs & Affiliations: Member of the Verduzco Polymer Engineering Laboratory at Rice. Collaborates with interdisciplinary teams on environmental and biomedical material innovations.
Els Henckaerts is a Professor at KU Leuven's Faculty of Medicine, Department of Cellular and Molecular Medicine, where she serves as head of the Trellis Research Group and Virus-Host Interactions and Therapeutic Approaches (VITA) Research Group. She additionally holds leadership roles as division head of the Virology Division Group 4 – Rega and is an active member of the KU Leuven Brain Institute and Leuven Institute for Rare Diseases. Her research integrates virology, molecular biology, and gene therapy with emphasis on adeno-associated virus (AAV) vector development for rare genetic disorders. Current investigations focus on dual AAV intein-based systems for DFNB9 deafness, nanobody-conjugated vectors for enhanced targeting specificity, and preclinical Parkinson's disease therapies. Her work bridges fundamental viral mechanisms with translational applications in inherited sensory and neurodegenerative conditions. Analysis of recent publications reveals dominant themes in AAV vector engineering, analytical characterization methods, and rare disease applications. Key trends include standardization of rAAV production processes, novel conjugation technologies for tissue-specific delivery, and advanced quantification techniques using digital droplet PCR and nanopore sequencing. Henckaerts leads multiple major initiatives including the European Research Alliance for Rare Diseases (2024-2031), an integrated AAV therapy development ecosystem (2025-2030), and the Gene Therapy Innovation Training Network (GET-IN). Her grant portfolio demonstrates significant funding for translational gene therapy projects with clinical endpoints. The Trellis Research Group operates across KU Leuven's Herestraat and Gaston Geenslaan campuses, maintaining specialized facilities for vector production, preclinical testing, and analytical characterization. The team collaborates extensively with clinical partners through the Rega Institute and participates in European consortia focused on rare disease therapeutics.
Michael A. Barry, Ph.D., is a Professor of Medicine at Mayo Clinic in Rochester, Minnesota, where he holds primary and joint appointments as a Consultant in the Department of Internal Medicine (Division of Infectious Diseases), Department of Immunology, and Department of Molecular Medicine. He leads the Virology, Vector and Vaccine Engineering Laboratory, focusing on developing advanced gene therapies, viral vectors, and vaccines for challenging diseases. Institution: Mayo Clinic School: College of Medicine and Science Department: Department of Internal Medicine Academic Rank: Professor Email: barry.michael@mayo.edu Education: Ph.D., Pharmacology and Toxicology, Dartmouth College Postgraduate Trainee, University of Texas Southwestern Medical Center B.S., Chemistry, Nebraska Wesleyan University Dr. Barry’s research centers on virology, gene therapy, and vaccine engineering. His lab develops in vivo molecular and viral therapies using adenovirus, adeno-associated virus (AAV), and lipid nanoparticles. Key areas include gene therapy for metabolic diseases like propionic acidemia and Alport syndrome, gene-based vaccines for HIV, influenza, Zika, and SARS-CoV-2, and oncolytic immunotherapy viruses for cancer. His team engineered a single-cycle adenovirus COVID-19 vaccine tested in Phase 1 trials (NCT04839042) and is advancing CRAd657-CD40L for melanoma clinical trials in 2025. They also work on basic virology of Ebola and pandemic influenza, leveraging findings for therapeutic development. His recent publications highlight innovations in adenoviral vector generation (FastAd toolkit), mucosal vaccine delivery, structural insights into adenovirus-blood interactions, and AAV-mediated therapies for musculoskeletal and gastrointestinal conditions. These works reflect a strong trend in translational virology and targeted therapeutics. Scientific Awards: None explicitly mentioned in the provided text. Advising and Grants: Dr. Barry has secured substantial grant funding, including from the National Institute of Allergy and Infectious Diseases (NIAID) and Congressionally Directed Medical Research Programs, for projects such as single-cycle SARS-CoV-2 vaccines, oncolytic therapies for kidney cancer, Ebola virus pathogenesis, mucosal HIV vaccines, and Zika virus vaccines. He mentors trainees and supports postdoctoral fellowships, though specific student names are not listed. His lab fosters collaboration across Mayo Clinic centers, including the Center for Individualized Medicine, Center for Regenerative Biotherapeutics, and Mayo Clinic Comprehensive Cancer Center. Labs and Teams: He directs the Virology, Vector and Vaccine Engineering Laboratory, which focuses on cell-targeted delivery, vector purification, mucosal vaccination, polymer shielding of vectors, and optical imaging for tracking. The lab employs high-throughput screening, genetic engineering, and animal imaging to optimize vector specificity and reduce off-target effects.
Pablo Perez Pinera is an Associate Professor in the Department of Bioengineering at the University of Illinois, with additional affiliations in Biomedical and Translational Sciences, Molecular and Integrative Physiology, and the Carl R. Woese Institute for Genomic Biology. His work focuses on advancing genome editing technologies, particularly CRISPR-based systems and base editing tools. Research interests include developing precision gene therapies, optimizing viral vector delivery for in vivo applications, and understanding how chromatin structure influences editing efficiency. Recent contributions highlight the creation of the SPLICER base editing toolbox for therapeutic exon skipping and advancements in protein engineering for next-generation genome editors. His articles emphasize innovations in genome editing applications, such as reducing Alzheimer’s disease precursors in mice via gene editing. Collaborations span interdisciplinary teams addressing biomedical challenges through molecular and genetic approaches.
