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Expanding and extending gene therapy of monogenic diseases of the haematopoietic system

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Publications

Gene Therapy for β-Hemoglobinopathies

Author(s): Marina Cavazzana, Chiara Antoniani, Annarita Miccio
Published in: Molecular Therapy, Issue 25/5, 2017, Page(s) 1142-1154, ISSN 1525-0016
DOI: 10.1016/j.ymthe.2017.03.024

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

Author(s): Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J. Cradick, Ante S. Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Published in: Blood, Issue 131/17, 2018, Page(s) 1960-1973, ISSN 0006-4971
DOI: 10.1182/blood-2017-10-811505

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype


Published in: ISSN 2329-0501
DOI: 10.1016/j.omtm.2018.07.012