| Year |
Citation |
Score |
| 2023 |
Andreoletti G, Romano O, Chou HJ, Sefid-Dashti MJ, Grilli A, Chen C, Lakshman N, Purushothaman P, Varfaj F, Mavilio F, Bicciato S, Urbinati F. High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy. American Journal of Human Genetics. PMID 37673065 DOI: 10.1016/j.ajhg.2023.08.008 |
0.303 |
|
| 2019 |
Zhu Y, Smith DJ, Zhou Y, Li YR, Yu J, Lee D, Wang YC, Di Biase S, Wang X, Hardoy C, Ku J, Tsao T, Lin LJ, Pham AT, Moon H, ... ... Urbinati F, et al. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer. Cell Stem Cell. PMID 31495780 DOI: 10.1016/J.Stem.2019.08.004 |
0.686 |
|
| 2018 |
Poletti V, Urbinati F, Charrier S, Corre G, Hollis RP, Campo Fernandez B, Martin S, Rothe M, Schambach A, Kohn DB, Mavilio F. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. Molecular Therapy. Methods & Clinical Development. 11: 167-179. PMID 30533448 DOI: 10.1016/J.Omtm.2018.10.014 |
0.729 |
|
| 2018 |
Urbinati F, Campo Fernandez B, Masiuk KE, Poletti V, Hollis RP, Koziol C, Kaufman M, Brown D, Mavilio F, Kohn DB. Gene Therapy for Sickle Cell Disease: a lentiviral vector comparison study. Human Gene Therapy. PMID 30198339 DOI: 10.1089/Hum.2018.061 |
0.737 |
|
| 2017 |
Urbinati F, Wherley J, Geiger S, Fernandez BC, Kaufman ML, Cooper A, Romero Z, Marchioni F, Reeves L, Read E, Nowicki B, Grassman E, Viswanathan S, Wang X, Hollis RP, et al. Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease. Cytotherapy. PMID 28733131 DOI: 10.1016/J.Jcyt.2017.06.002 |
0.705 |
|
| 2017 |
Masiuk KE, Brown D, Laborada J, Hollis RP, Urbinati F, Kohn DB. Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28663101 DOI: 10.1016/J.Ymthe.2017.05.023 |
0.702 |
|
| 2016 |
Bjurström CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP, Kohn DB. Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. Molecular Therapy. Nucleic Acids. 5: e351. PMID 28131278 DOI: 10.1038/Mtna.2016.52 |
0.715 |
|
| 2016 |
DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE. Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Science Translational Medicine. 8: 360ra134. PMID 27733558 DOI: 10.1126/Scitranslmed.Aaf9336 |
0.715 |
|
| 2016 |
Hoban MD, Lumaquin D, Kuo CY, Romero Z, Long J, Ho M, Young CS, Mojadidi M, Fitz-Gibbon S, Cooper AR, Lill GR, Urbinati F, Campo-Fernandez B, Bjurstrom CF, Pellegrini M, et al. CRISPR/Cas9-mediated correction of the sickle mutation in human CD34+ cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 27406980 DOI: 10.1038/Mt.2016.148 |
0.716 |
|
| 2016 |
Masiuk KE, Brown D, Urbinati F, Hollis RP, Kohn DB. Enrichment of Hematopoietic Stem Cells Using Immunomagnetic Beads to Facilitate Gene Therapy Blood. 128: 2313-2313. DOI: 10.1182/Blood.V128.22.2313.2313 |
0.699 |
|
| 2016 |
Urbinati F, Poletti V, Fernandez BC, Hollis RP, Koziol C, Kaufman ML, Brown D, Miccio A, Antoniou M, Kohn DB, Mavilio F. 230. Designing High-Titer Lentiviral Vectors for Gene Therapy of Sickle-Cell Disease Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33039-8 |
0.747 |
|
| 2015 |
Romero Z, Campo-Fernandez B, Wherley J, Kaufman ML, Urbinati F, Cooper AR, Hoban MD, Baldwin KM, Lumaquin D, Wang X, Senadheera S, Hollis RP, Kohn DB. The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. Molecular Therapy. Methods & Clinical Development. 2: 15012. PMID 26029723 DOI: 10.1038/Mtm.2015.12 |
0.707 |
|
| 2015 |
Hoban MD, Cost GJ, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu PQ, et al. Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood. 125: 2597-604. PMID 25733580 DOI: 10.1182/Blood-2014-12-615948 |
0.739 |
|
| 2015 |
Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A. Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells. Experimental Hematology. 43: 346-51. PMID 25681747 DOI: 10.1016/J.Exphem.2015.01.009 |
0.749 |
|
| 2015 |
Baldwin K, Urbinati F, Romero Z, Campo-Fernandez B, Kaufman ML, Cooper AR, Masiuk K, Hollis RP, Kohn DB. Enrichment of human hematopoietic stem/progenitor cells facilitates transduction for stem cell gene therapy. Stem Cells (Dayton, Ohio). 33: 1532-42. PMID 25588820 DOI: 10.1002/Stem.1957 |
0.687 |
|
| 2015 |
Hoban MD, Mendel MC, Romero Z, Kaufman ML, Joglekar AV, Ho M, Lumaquin D, Gray D, Lill GR, Cooper AR, Urbinati F, Senadheera S, Zhu A, Liu P, Paschon DE, et al. 115. Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33720-0 |
0.673 |
|
| 2013 |
Romero Z, Urbinati F, Geiger S, Cooper AR, Wherley J, Kaufman ML, Hollis RP, de Assin RR, Senadheera S, Sahagian A, Jin X, Gellis A, Wang X, Gjertson D, Deoliveira S, et al. β-globin gene transfer to human bone marrow for sickle cell disease. The Journal of Clinical Investigation. PMID 23863630 DOI: 10.1172/Jci67930 |
0.75 |
|
| 2013 |
Hoban MD, Joglekar AV, Gray D, Kaufman ML, Urbinati F, Senadheera S, Cost G, Reik A, Holmes MC, Gregory PD, Hollis RP, Kohn DB. Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34+ Cells Blood. 122: 2904-2904. DOI: 10.1182/Blood.V122.21.2904.2904 |
0.736 |
|
| 2012 |
Baldwin KM, Urbinati F, Romero-Garcia Z, Kohn DB. Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy Blood. 120: 2052-2052. DOI: 10.1182/Blood.V120.21.2052.2052 |
0.71 |
|
| 2011 |
Liu R, Zheng L, Dwyre DM, Urbinati F, Kohn DB, Matthews D, Chan J, Satake N. Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease Blood. 118: 4847-4847. DOI: 10.1182/Blood.V118.21.4847.4847 |
0.633 |
|
| 2011 |
Urbinati F, Garcia ZR, Geiger S, de Assin RR, Kuftinec G, Hollis RP, Oliveira SND, Wherley J, Cooper A, Townes TM, Crooks GM, Read EJ, Walters MC, Meiselman HJ, Coates TD, et al. Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector Blood. 118: 3119-3119. DOI: 10.1182/Blood.V118.21.3119.3119 |
0.766 |
|
| 2009 |
Arumugam PI, Higashimoto T, Urbinati F, Modlich U, Nestheide S, Xia P, Fox C, Corsinotti A, Baum C, Malik P. Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1929-1937. PMID 28178459 DOI: 10.1038/mt.2009.183 |
0.574 |
|
| 2009 |
Arumugam PI, Urbinati F, Velu CS, Higashimoto T, Grimes HL, Malik P. The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity. Plos One. 4: e6995. PMID 19746166 DOI: 10.1371/Journal.Pone.0006995 |
0.579 |
|
| 2009 |
Arumugam PI, Higashimoto T, Urbinati F, Modlich U, Nestheide S, Xia P, Fox C, Corsinotti A, Baum C, Malik P. Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1929-37. PMID 19707188 DOI: 10.1038/Mt.2009.183 |
0.604 |
|
| 2009 |
Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D, Malik P. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood. 114: 1174-85. PMID 19474450 DOI: 10.1182/Blood-2009-01-201863 |
0.666 |
|
| 2009 |
Urbinati F, Arumugam P, Higashimoto T, Perumbeti A, Mitts K, Xia P, Malik P. Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1527-36. PMID 19384292 DOI: 10.1038/Mt.2009.89 |
0.603 |
|
| 2009 |
Arumugam P, Urbinati F, Velu CS, Higashimoto T, Grimes HL, Malik P. A Novel Combination of Chicken Hypersensitive Site-4 Insulator Elements Improves Titers and Restores Full Insulator Activity. Blood. 114: 3566-3566. DOI: 10.1182/Blood.V114.22.3566.3566 |
0.581 |
|
| 2008 |
Arumugam P, Urbinati F, Velu CS, Grimes HL, Malik P. The 3′ End of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to the 5′ Insulator Core and Is Necessary in Conjunction with the Core for Full Insulator Activity Blood. 112: 817-817. DOI: 10.1182/Blood.V112.11.817.817 |
0.498 |
|
| 2008 |
Urbinati F, Arumugam P, Higashimoto T, Perumbeti A, Xia P, Malik P. Mechanism of Reduction in Titers from Lentivirus Vectors Carrying Chromatin Insulator Elements in the 3′ LTR Blood. 112: 2359-2359. DOI: 10.1182/Blood.V112.11.2359.2359 |
0.607 |
|
| 2008 |
Perumbeti A, Higashimoto T, Urbinati F, Lauderback K, Loberg A, Estevez-Pagani G, Meiselman H, Franco R, Witte D, Joiner CH, Malik P. Correction of Sickle Cell Anemia with γ-Globin Gene Delivered by Lentivirus Vector in the Setting of Myeloablative or Reduced Intensity Conditioning, and Establishing Critical Determinants for Successful Gene Therapy for Sickle Cell Disease. Blood. 112: 2354-2354. DOI: 10.1182/Blood.V112.11.2354.2354 |
0.609 |
|
| 2008 |
Arumugam P, Urbinati F, Higashimoto T, Perumbeti A, Malik P. Lentivirus vectors for gene therapy for hemoglobinopathies Blood Cells, Molecules, and Diseases. 40: 275-276. DOI: 10.1016/J.Bcmd.2007.10.059 |
0.58 |
|
| 2008 |
Arumugam P, Xia P, Urbinati F, Malik P. Elements of cHS4 insulator necessary for maximum chromatin barrier activity Blood Cells, Molecules, and Diseases. 40: 252. DOI: 10.1016/J.Bcmd.2007.10.015 |
0.461 |
|
| 2007 |
Aiuti A, Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Aker M, Slavin S, Cazzola M, Sartori D, Ambrosi A, Di Serio C, et al. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. The Journal of Clinical Investigation. 117: 2233-40. PMID 17671653 DOI: 10.1172/Jci31666 |
0.572 |
|
| 2007 |
Higashimoto T, Urbinati F, Perumbeti A, Jiang G, Zarzuela A, Chang LJ, Kohn DB, Malik P. The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors. Gene Therapy. 14: 1298-304. PMID 17597793 DOI: 10.1038/sj.gt.3302979 |
0.656 |
|
| 2007 |
Arumugam PI, Xia P, Urbinati F, Malik P. Elements of a Chromatin Insulator Necessary for Consistent, High-Level Globin Gene Expression Blood. 110: 3731-3731. DOI: 10.1182/Blood.V110.11.3731.3731 |
0.513 |
|
| 2006 |
Urbinati F, Madigan C, Malik P. Pathophysiology and therapy for haemoglobinopathies. Part II: thalassaemias. Expert Reviews in Molecular Medicine. 8: 1-26. PMID 16684395 DOI: 10.1017/S1462399406010805 |
0.519 |
|
| 2006 |
Recchia A, Bonini C, Magnani Z, Urbinati F, Sartori D, Muraro S, Tagliafico E, Bondanza A, Stanghellini MT, Bernardi M, Pescarollo A, Ciceri F, Bordignon C, Mavilio F. Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells. Proceedings of the National Academy of Sciences of the United States of America. 103: 1457-62. PMID 16432223 DOI: 10.1182/Blood.V106.11.462.462 |
0.515 |
|
| 2006 |
Cassani B, Andolfi G, Mirolo M, Biasco L, Recchia A, Urbinati F, Valacca C, Scaramuzza S, Cazzola M, Sartori D, Ambrosi A, Serio CD, Roncarolo MG, Mavilio F, Bordignon C, et al. Clonal Analyses of Retroviral Vector Integrations in ADA-SCID Patients Treated with Stem Cell Gene Therapy. Blood. 108: 3249-3249. DOI: 10.1182/Blood.V108.11.3249.3249 |
0.548 |
|
| 2006 |
Urbinati F, Xia P, Perumbeti A, Xia M, Corsinotti A, Zarzuela A, Perelman N, Malik P. 467. Lentiviral Vector (LV) Non-Coding Elements That Impart High Titers and Expression to β-Globin (hβ) Cassettes Molecular Therapy. 13: S181. DOI: 10.1016/J.Ymthe.2006.08.536 |
0.574 |
|
| 2005 |
Urbinati F, Lotti F, Facchini G, Montanari M, Ferrari G, Mavilio F, Grande A. Competitive engraftment of hematopoietic stem cells genetically modified with a truncated erythropoietin receptor. Human Gene Therapy. 16: 594-608. PMID 15916484 DOI: 10.1089/Hum.2005.16.594 |
0.515 |
|
| 2005 |
Recchia A, Magnani Z, Urbinati F, Sartori D, Muraro S, Stanghellini MTL, Bernardi M, Pescarollo A, Bregni M, Bonini C, Bordignon C, Ciceri F, Mavilio F. 819. Molecular Follow-Up of Patients Treated with Allogeneic Hematopoietic Stem Cell Transplantation and Donor Lymphocytes Transduced with a Retroviral Vector Expressing HSV-TK and |[Delta]|LNGFR Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.362 |
0.533 |
|
| 2004 |
Bonini C, Magnani Z, Recchia A, Urbinati F, Muraro S, Tech SS, Ciceri F, Bernardi M, Pescarollo A, Gallo-Stampino C, Bregni M, Bordignon C, Mavilio F. Molecular Follow-Up of Patients Treated with HSV-TK/ΔLNGFR Engineered Donor Lymphocytes after Allogeneic Stem Cell Transplantation. Blood. 104: 1741-1741. DOI: 10.1182/Blood.V104.11.1741.1741 |
0.543 |
|
| 2004 |
Urbinati F, Grande A, Lotti F, Facchini G, Ferrari G, Mavilio F. 403. Selective Engraftment of Genetically Modified Hematopoietic Stem Cells by a Truncated Erythropoietin Receptor Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.360 |
0.533 |
|
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