Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapyMaxwell Chappell, Stefano Rivella, Valentina Ghiaccio et al.|Blood|2024Cited by 11
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
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
Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-ThalassemiaMaxwell Chappell, Stefano Rivella, Laura Breda et al.|Blood|2022Cited by 0