Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapyMaxwell Chappell, Stefano Rivella, Kandace Gollomp et al.|Blood|2024Cited by 11
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
A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its TreatmentMaxwell Chappell, Stefano Rivella, Danuta Jarocha et al.|Blood|2021Cited by 1
P1521: A SEVERE MOUSE MODEL OF ALPHA-THALASSEMIA SHOWS ABNORMAL IRON METABOLISM, ERYTHROPOIESIS AND COAGULATION, AND CAN BE RESCUED BY A NOVEL GENE THERAPY APPROACHStefano Rivella, Osheiza Abdulmalik, Maxwell Chappell et al.|HemaSphere|2022Cited by 1
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