Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathiesLaura Breda, Stefano Rivella, Alisa Dong et al.|Molecular Therapy|2021Cited by 23
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
2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential TherapiesValentina Ghiaccio, Laura Breda, Alisa Dong et al.|Blood|2019Cited by 0