Overcoming preexisting humoral immunity to AAV using capsid decoys F Mingozzi, XM Anguela, G Pavani, Y Chen, RJ Davidson, DJ Hui, ... Science translational medicine 5 (194), 194ra92-194ra92, 2013 | 354 | 2013 |
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus C Antoniani, V Meneghini, A Lattanzi, T Felix, O Romano, E Magrin, ... Blood, The Journal of the American Society of Hematology 131 (17), 1960-1973, 2018 | 215 | 2018 |
Optimization of CRISPR/Cas9 delivery to human hematopoietic stem and progenitor cells for therapeutic genomic rearrangements A Lattanzi, V Meneghini, G Pavani, F Amor, S Ramadier, T Felix, ... Molecular Therapy 27 (1), 137-150, 2019 | 128 | 2019 |
Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions E Masat, G Pavani, F Mingozzi Discovery medicine 15 (85), 379-389, 2013 | 115 | 2013 |
A zymogen-like factor Xa variant corrects the coagulation defect in hemophilia L Ivanciu, R Toso, P Margaritis, G Pavani, H Kim, A Schlachterman, JH Liu, ... Nature biotechnology 29 (11), 1028-1033, 2011 | 102 | 2011 |
In vivo hematopoietic stem cell modification by mRNA delivery L Breda, TE Papp, MP Triebwasser, A Yadegari, MT Fedorky, N Tanaka, ... Science 381 (6656), 436-443, 2023 | 93 | 2023 |
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells G Pavani, A Fabiano, M Laurent, F Amor, E Cantelli, A Chalumeau, ... Blood advances 5 (5), 1137-1153, 2021 | 66 | 2021 |
Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins G Pavani, M Laurent, A Fabiano, E Cantelli, A Sakkal, G Corre, PJ Lenting, ... Nature communications 11 (1), 3778, 2020 | 39 | 2020 |
Targeted gene delivery: where to land G Pavani, M Amendola Frontiers in genome editing 2, 609650, 2021 | 38 | 2021 |
The protein C pathway and sepsis P Della Valle, G Pavani, A D'Angelo Thrombosis research 129 (3), 296-300, 2012 | 37 | 2012 |
Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII OA Marcos-Contreras, SM Smith, DA Bellinger, RA Raymer, E Merricks, ... Blood, The Journal of the American Society of Hematology 127 (5), 565-571, 2016 | 31 | 2016 |
The endothelial protein C receptor enhances hemostasis of FVIIa administration in hemophilic mice in vivo G Pavani, L Ivanciu, A Faella, OA Marcos-Contreras, P Margaritis Blood, The Journal of the American Society of Hematology 124 (7), 1157-1165, 2014 | 31 | 2014 |
Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo P Margaritis, E Roy, A Faella, HD Downey, L Ivanciu, G Pavani, S Zhou, ... Blood, The Journal of the American Society of Hematology 117 (15), 3974-3982, 2011 | 22 | 2011 |
Study of inherited thrombocytopenia resulting from mutations in ETV6 or RUNX1 using a human pluripotent stem cell model S Borst, CC Nations, JG Klein, G Pavani, JA Maguire, RM Camire, ... Stem Cell Reports 16 (6), 1458-1467, 2021 | 18 | 2021 |
The use of pluripotent stem cells to generate diagnostic tools for transfusion medicine HH An, AL Gagne, JA Maguire, G Pavani, O Abdulmalik, P Gadue, ... Blood, The Journal of the American Society of Hematology 140 (15), 1723-1734, 2022 | 13 | 2022 |
Membrane binding and anticoagulant properties of protein S natural variants M Baroni, G Pavani, D Marescotti, T Kaabache, D Borgel, S Gandrille, ... Thrombosis Research 125 (2), e33-e39, 2010 | 12 | 2010 |
Asymmetric processing of mutant factor X Arg386Cys reveals differences between intrinsic and extrinsic pathway activation M Baroni, G Pavani, M Pinotti, A Branchini, F Bernardi, RM Camire Biochimica et Biophysica Acta (BBA)-Proteins and Proteomics 1854 (10), 1351-1356, 2015 | 11 | 2015 |
Gene-based FVIIa prophylaxis modulates the spontaneous bleeding phenotype of hemophilia A rats SM Zintner, JC Small, G Pavani, L Dankner, OA Marcos-Contreras, ... Blood Advances 3 (3), 301-311, 2019 | 6 | 2019 |
One amino acid in mouse activated factor VII defines its endothelial protein C receptor (EPCR) binding and modulates its EPCR‐dependent hemostatic activity in vivo G Pavani, SM Zintner, L Ivanciu, JC Small, KA Stafford, JH Szeto, ... Journal of Thrombosis and Haemostasis 15 (3), 507-512, 2017 | 6 | 2017 |
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated disruption of the β-globin locus architecture C Antoniani, V Meneghini, A Lattanzi, G Pavani, T Felix, F Amor, ... Blood 128 (22), 321, 2016 | 5 | 2016 |