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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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Targeted Cargo Delivery to Mouse Lower Limb by Exosomes Carrying a Muscle Targeting Moiety

Minghao Sun, Mafalda Cacciottolo, Yujia Li, Firouz Moshenian, Meena Murali, William Morrison, Michael LeClaire, Kristi Elliott

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Exosomes loaded with siRNA targeting the RRM2 gene trigger apoptosis in the SKOV3 Ovarian Cancer cell-line

Firouz Mohsenian, Meena Murali, Michael LeClaire, Cindy Lau, Kristi Elliot, Minghao Sun

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Long Term Safety and Efficacy of CAP-1002 in late-stage patients with DMD: A New Treatment Approach to Target Skeletal and Cardiac Muscle Pathogenesis (24-Month Data from HOPE-2-OLE Study)

Craig M. McDonald, MD, Suzanne Hendrix, PhD, Michelle Eagle, PhD, Matthew M. Harmelink, MD, Arun S. Varadhachary, MD, PhD, Cuixia Tian, MD, Susan Apkon, MD, Chet Villa, MD, Michael D. Taylor, MD, Kan N. Hor, MD, Mitchell Wassom, MS, Upagna Desai, Mark S. Awadalla, Linda Marbán, PhD

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Targeted ASO Delivery To Mouse Lower Limb by Exosome Carrying A Muscle Targeting Moiety

Minghao Sun, Yujia Li, SunMafalda Cacciottolo, Mahrou Sadri, Michael LeClaire, David Tran, Kristi Elliott,, Linda Marbán

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Exosomes: Scalable Production, Purification, and Characterization Methods using 293F Cells

Michael LeClaire, Ph.D, Justin Nice, Ph.D, Yujia Li, Ph.D, Christy Lau, Stephanie Adachi, Ciana Mora, Mafalda Cacciottolo, Ph.D, Kristi Elliott, Ph.D, Minghao Sun, Ph.D

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Nanograms of SARS-CoV-2 spike protein delivered by exosomes induce potent neutralization of both delta and omicron variants

Mafalda Cacciottolo, Yujia Li, Justin B. Nice, Michael J. LeClaire, Ryan Twaddle, Ciana L. Mora, Stephanie Y. Adachi, Meredith Young, Jenna Angeles, Kristi Elliott, Minghao Sun*

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