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Extracellular Vesicles Delivered a Functional ARG1 Enzyme and Restored Its Activity in a Mouse Model of ARG1-D Resulting in Improved Lifespan

Li-En Hsieh, Mafalda Cacciottolo, Michael J. LeClaire, William Morrison, Bailey Murphy, Christy Lau, Kristi Elliott, Linda Marban and Minghao Sun

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StealthX: A Versatile and Potent Exosome-Based Vaccine Platform for the Next Pandemic

Minghao Sun, Kristi Elliott

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A Systematic Framework for the Scalable Loading of Therapeutic siRNA and PMO into Exosomes

Firouz Mohsenian, Meena Murali, David Tran, Daniel Leychkis, William Morrison, Kristi Elliott, Minghao Sun

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A Novel In Vitro Potency Assay Demonstrating the Anti-Fibrotic Mechanism of Action of CDCs in Deramiocel

Yujia Li, Justin B. Nice, Marya Kozinova , Stephanie Adachi, Linda Marban, Kristi Elliott and Minghao Sun

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Multivalent Exosome Based Protein Vaccine: A “Mix and Match” Approach to Epidemic Viruses’ Challenges

Mafalda Cacciottolo, Li-En Hsieh, Yujia Li, Michael J. LeClaire, Ciana L. Mora, Christy Lau, Charles Dwyer, Kristi Elliott and Minghao Sun

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Treatment with Deramiocel in DMD: Evidence of Long-Term Stabilization in Performance of Upper Limb Function Compared to Matched External Comparators (MDA 2025)

Craig M. McDonald, M.D., Chet Villa, M.D., Cuixia Tian, M.D., Alexander M. Zygmunt, M.D., Nicholas J. Ollberding, Ph.D., Kevin Berth, Upagna Desai, Zena Askerova Ph.D., Nathaniel J. Hogan, Ph.D., Kati S. Maharry, Ph.D., Mark S. Awadalla, Linda Marbán, Ph.D.

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Engineered exosomes loaded with phosphorodiamidate morpholino oligomers targeting mouse exon 23 promote exon skipping in-vitro and in-vivo

Firouz Mohsenian, Meena Murali, Michael LeClaire, Christy Lau, Mafalda Cacciottolo, Kristi Elliott, Minghao Sun

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Multi-modal benefits of deramiocel (CAP-1002) in late-stage patients with DMD: a new treatment approach to target skeletal and cardiac muscle pathogenesis (HOPE 2-OLE trial: 36-month data) (WMS 2024)

Craig M. McDonald, M.D., Chet Villa, M.D., Jonathan H. Soslow, M.D., Cuixia Tian, M.D., Alexander M. Zygmunt, M.D., Nicholas J. Ollberding, Ph.D., Kan N. Hor, M.D., Matthew M. Harmelink, M.D., Arun S. Varadhachary, M.D., Ph.D., Susan Apkon, M.D., Veena Ramanna, Upagna Desai, Nathaniel J. Hogan, Ph.D., Kati S. Maharry, Ph.D., Mark S. Awadalla, Linda Marbán, Ph.D.

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Deramiocel (CAP-1002) Demonstrates Disease Modification in Later-Stage DMD Patients 36-Month Safety and Efficacy Results from the HOPE-2 Open Label Extension (PPMD presentation)

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Exosome-Mediated Intracellular Delivery of Arginase 1: a Potential Application for the Treatment of Arginase-1 Deficiency

Li-En Hsieh, Mafalda Cacciottolo, Michael LeClaire, Christy Lau, Kristi Elliott, Minghao Sun

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