| A comprehensive single cell transcriptional landscape of human hematopoietic progenitors D Pellin, M Loperfido, C Baricordi, SL Wolock, A Montepeloso, ... Nature communications 10 (1), 2395, 2019 | 285 | 2019 |
| Efficient derivation and inducible differentiation of expandable skeletal myogenic cells from human ES and patient-specific iPS cells SM Maffioletti, MFM Gerli, M Ragazzi, S Dastidar, S Benedetti, ... Nature protocols 10 (7), 941-958, 2015 | 107 | 2015 |
| Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases F Daboussi, M Zaslavskiy, L Poirot, M Loperfido, A Gouble, V Guyot, ... Nucleic acids research 40 (13), 6367-6379, 2012 | 106 | 2012 |
| Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome S Scaramuzza, L Biasco, A Ripamonti, MC Castiello, M Loperfido, ... Molecular Therapy 21 (1), 175-184, 2013 | 97 | 2013 |
| Next-generation muscle-directed gene therapy by in silico vector design S Sarcar, W Tulalamba, MY Rincon, J Tipanee, HQ Pham, H Evens, ... Nature communications 10 (1), 492, 2019 | 46 | 2019 |
| piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts M Loperfido, S Jarmin, S Dastidar, M Di Matteo, I Perini, M Moore, N Nair, ... Nucleic acids research 44 (2), 744-760, 2016 | 35 | 2016 |
| Pluripotent stem cells for gene therapy of degenerative muscle diseases M Loperfido, H B Steele-Stallard, F Saverio Tedesco, T VandenDriessche Current gene therapy 15 (4), 364-380, 2015 | 29 | 2015 |
| High-throughput analysis of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing RN Plasschaert, MP DeAndrade, F Hull, Q Nguyen, T Peterson, A Yan, ... Molecular Therapy 30 (10), 3209-3225, 2022 | 8 | 2022 |
| IS-Seq: a bioinformatics pipeline for integration sites analysis with comprehensive abundance quantification methods A Yan, C Baricordi, Q Nguyen, L Barbarossa, M Loperfido, L Biasco BMC bioinformatics 24 (1), 286, 2023 | 1 | 2023 |
| Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations JK Yoon, JW Schindler, M Loperfido, C Baricordi, MP DeAndrade, ... bioRxiv, 2023.11. 03.565442, 2023 | | 2023 |
| High-Resolution Cellular and Molecular Follow-Up of Lysosomal Disease Patients Treated with Autologous Hematopoietic Stem Cell Lentiviral Gene Therapy M Loperfido, C Baricordi, A Yan, Q Nguyen, L Barbarossa, C Mason, ... MOLECULAR THERAPY 30 (4), 374-374, 2022 | | 2022 |
| High throughput monitoring of safety, potency and stability of gene therapy cell products in lysosomal disease patients L Biasco, C Baricordi, M Loperfido, A Yan, L Barbarossa, I Abrahamsen, ... Molecular Genetics and Metabolism 135 (2), S23, 2022 | | 2022 |
| High-resolution cellular and molecular follow up of lysosomal disease patients treated with hematopoietic stem cell lentiviral gene therapy M Loperfido, C Baricordi, A Yan, L Barbarossa, Q Nguyen, I Abrahamsen, ... Molecular Genetics and Metabolism 135 (2), S75-S76, 2022 | | 2022 |
| Compositions and methods for cell transplantation L Biasco, M Loperfido, C Baricordi, D Pellin US Patent App. 17/213,417, 2021 | | 2021 |
| High-resolution cellular and molecular follow-up of lysosomal disorder patients treated with hematopoietic stem cell lentiviral gene therapy M Loperfido, C Baricordi, A Yan, L Barbarossa, H Chamarthi, ... HUMAN GENE THERAPY 32 (19-20), A22-A22, 2021 | | 2021 |
| Transplantation of gene-modified haematopoietic stem cells and their application in murine models of neurodegenerative disease RN Plasschaert, MP DeAndrade, F Hull, Q Nguyen, T Peterson, ... HUMAN GENE THERAPY 32 (19-20), A49-A49, 2021 | | 2021 |
| High throughput characterization of CD34+ cell states and dynamics upon genetic engineering C Baricordi, M Loperfido, A Yan, H Chamarthi, L Barbarossa, A Golipour, ... HUMAN GENE THERAPY 32 (19-20), A140-A140, 2021 | | 2021 |
| Genetically engineered microglia-like cells have therapeutic potential for neurodegenerative disease RN Plasschaert, MP DeAndrade, F Hull, Q Nguyen, T Peterson, A Yan, ... bioRxiv, 2021.09. 16.460703, 2021 | | 2021 |
| A comprehensive characterization of biodistribution and single-cell states of HSC-derived microglia upon ex-vivo lentiviral gene therapy in murine models of neurological disease RN Plasschaert, M DeAndrade, M Loperfido, C Baricordi, L Barbarossa, ... 2021 Alzheimer's Association International Conference, 2021 | | 2021 |
| Analysis of genetically engineered stem cell product and follow up of gene therapy patients through high-throughput single cell technologies C Baricordi, M Loperfido, A Yan, H Chamarthi, L Barbarossa, A Golipour, ... Molecular Genetics and Metabolism 132 (2), S18, 2021 | | 2021 |
Cristina BaricordiBeam TherapeuticsVerified email at beamtx.com
