Corti, Manuela

Current clinical applications of in vivo gene therapy with AAVs

Mendell, J. R., Al-Zaidy, S. A., Rodino-Klapac, L. R., Goodspeed, K., Gray, S. J., Kay, C. N., Boye, S. L., Boye, S. E., George, L. A., Salabarria, S., et al. (2021) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

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Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

Salabarria, S. M., Corti, M., Coleman, K. E., Wichman, M. B., Berthy, J. A., D’Souza, P., Tifft, C. J., Herzog, R. W., Elder, M. E., Shoemaker, L. R., et al. (2024) · Journal of Clinical Investigation

Virus-based gene therapy researchComplement system in diseases

Cited by 159

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

Corti, M., Elder, M., Falk, D., Lawson, L., Smith, B., Nayak, S., Conlon, T., Clément, N., Erger, K., Lavassani, E., et al. (2014) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 140

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV- hGAA ) Gene Therapy in Children Affected by Pompe Disease

Corti, M., Liberati, C., Smith, B. K., Lawson, L. A., Tuna, I. S., Conlon, T. J., Coleman, K. E., Islam, S., Herzog, R. W., Fuller, D. D., et al. (2017) · Human Gene Therapy Clinical Development

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 120

Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases

Perez, B. A., Shutterly, A., Chan, Y. K., Byrne, B. J., & Corti, M. (2020) · Brain Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 112

Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy

Byrne, B. J., Fuller, D. D., Smith, B. K., Clement, N., Coleman, K., Cleaver, B., Vaught, L., Falk, D. J., McCall, A., & Corti, M. (2019) · Annals of Translational Medicine

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

Cited by 51

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver

Rana, J., Herzog, R. W., Muñoz-Melero, M., Yamada, K., Kumar, S. R. P., Lam, A. K., Markusic, D. M., Duan, D., Terhorst, C., Byrne, B. J., et al. (2024) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

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Considerations for Systemic Use of Gene Therapy

Byrne, B. J., Corti, M., & Muntoni, F. (2021) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 23

Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction: Neuromuscular Activation in Pompe

Corti, M., Smith, B. K., Falk, D. J., Lawson, L. A., Fuller, D. D., Subramony, S. H., Byrne, B. J., & Christou, E. A. (2015) · Muscle & Nerve

Lysosomal Storage Disorders ResearchMuscle Physiology and Disorders

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