Biffi, Alessandra

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., Baldoli, C., Martino, S., Calabria, A., Canale, S., et al. (2013) · Science

Virus-based gene therapy researchLysosomal Storage Disorders Research

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Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., Dionisio, F., Calabria, A., Giannelli, S., Castiello, M. C., et al. (2013) · Science

Virus-based gene therapy researchCAR-T cell therapy research

Cited by 1,025

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Wu, Y., Zeng, J., Roscoe, B. P., Liu, P., Yao, Q., Lazzarotto, C. R., Clement, K., Cole, M. A., Luk, K., Baricordi, C., et al. (2019) · Nature Medicine

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

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Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Sessa, M., Lorioli, L., Fumagalli, F., Acquati, S., Redaelli, D., Baldoli, C., Canale, S., Lopez, I. D., Morena, F., Calabria, A., et al. (2016) · The Lancet

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 502

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

Esrick, E. B., Lehmann, L. E., Biffi, A., Achebe, M., Brendel, C., Ciuculescu, M. F., Daley, H., MacKinnon, B., Morris, E., Federico, A., et al. (2021) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 449

A comprehensive single cell transcriptional landscape of human hematopoietic progenitors

Pellin, D., Loperfido, M., Baricordi, C., Wolock, S. L., Montepeloso, A., Weinberg, O. K., Biffi, A., Klein, A. M., & Biasco, L. (2019) · Nature Communications

Hematopoietic Stem Cell TransplantationSingle-cell and spatial transcriptomics

Cited by 360

The changing face of adrenoleukodystrophy

Zhu, J., Eichler, F., Biffi, A., Duncan, C. N., Williams, D. A., & Majzoub, J. A. (2020) · Endocrine Reviews

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

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Microglial dysfunction as a key pathological change in adrenomyeloneuropathy

Gong, Y., Sasidharan, N., Laheji, F., Frosch, M., Musolino, P., Tanzi, R., Kim, D. Y., Biffi, A., El Khoury, J., & Eichler, F. (2017) · Annals of Neurology

Neuroinflammation and Neurodegeneration MechanismsLysosomal Storage Disorders Research

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