Miccio, Annarita

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

Cited by 194

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

Gene therapy of hemoglobinopathies: progress and future challenges

Ikawa, Y., Miccio, A., Magrin, E., Kwiatkowski, J. L., Rivella, S., & Cavazzana, M. (2019) · Human Molecular Genetics

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

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Sickle Cell Disease: From Genetics to Curative Approaches

Hardouin, G., Magrin, E., Corsia, A., Cavazzana, M., Miccio, A., & Semeraro, M. (2023) · Annual Review of Genomics and Human Genetics

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 40