Weber, Leslie

Gene therapy in a patient with sickle cell disease

Ribeil, J.-A., Hacein-Bey-Abina, S., Payen, E., Magnani, A., Semeraro, M., Magrin, E., Caccavelli, L., Neven, B., Bourget, P., El Nemer, W., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Antoniani, C., Meneghini, V., Lattanzi, A., Felix, T., Romano, O., Magrin, E., Weber, L., Pavani, G., El Hoss, S., Kurita, R., et al. (2018) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

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Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Weber, L., Frati, G., Felix, T., Hardouin, G., Casini, A., Wollenschlaeger, C., Meneghini, V., Masson, C., De Cian, A., Chalumeau, A., et al. (2020) · Science Advances

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 140

A microglia clonal inflammatory disorder in Alzheimer’s disease

Vicario, R., Fragkogianni, S., Weber, L., Lazarov, T., Hu, Y., Hayashi, S. Y., Craddock, B., Socci, N. D., Alberdi, A., Baako, A., et al. (2025) · eLife

Neuroinflammation and Neurodegeneration MechanismsAlzheimer's disease research and treatments

Cited by 21