Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathiesLaura Breda, Stefano Rivella, Virginia Guzikowski et al.|Molecular Therapy|2021Cited by 23
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Inclusion of a short hairpin RNA targeting <i>BCL11A</i> into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobinSílvia Pires Lourenço, Stefano Rivella, Danuta Jarocha et al.|Haematologica|2021Cited by 10
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalanceAlisa Dong, Laura Breda, Valentina Ghiaccio et al.|Haematologica|2020Cited by 2
PP-14, a Novel Structurally-Enhanced Antisickling Allosteric Hemoglobin Effector, Increases Oxygen Affinity and Disrupts Hemoglobin S Polymer FormationMartin K. Safo, Osheiza Abdulmalik, Piyusha P. Pagare et al.|Blood|2019Cited by 1