Research Focus

Research Facilities; NE Affiliate

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1. Early time post-vector activation of complement by AAV vectors in nonhuman primates

   Meeting Abstract

   Rosenberg, J.; De, B.; Caragiulo, A.; Kaminsky, S.; Martin, H.; Butler, S.; Rodo, J.; Sondhi, D.; Crystal, R.
   Molecular Therapy (2025)

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2. Safety of ascending doses of AAVrh.10hFXN to treat the cardiac manifestations of Friedreich's ataxia

   Meeting Abstract

   Rosenberg, J. B.; Greco, A.; Zuluaga, C. M.; Gertz, M. L. R, ; Yost-Bido, M.; Gorman, N. C.; Chen, A.; Kooner, V.; De, B. P.; Kaminsky, S. M.; more...
   Molecular Therapy (2023)

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3. Identification of safe and effective intravenous dose of AAVrh.10hFXN to treat the cardiac manifestations of Friedreich's ataxia

   Journal Article

   Munoz-Zuluaga, C.; Gertz, M.; Yost-Bido, M.; Greco, A.; Gorman, N.; Chen, A.; Kooner, V.; Rosenberg, J. B.; De, B. P.; Kaminsky, S. M.; more...
   Human Gene Therapy (2023)

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