Francesca Ficara

@article{pmid31938717,

title = {Generation of an immunodeficient mouse model of tcirg1-deficient autosomal recessive osteopetrosis},

author = {E Palagano and S Muggeo and L Crisafulli and I L Tourkova and D Strina and S Mantero and E Fontana and S L Locatelli and M Monari and E Morenghi and C Carlo-Stella and J B Barnett and H C Blair and P Vezzoni and A Villa and C Sobacchi and F Ficara},

year  = {2020},

date = {2020-06-01},

journal = {Bone Rep},

volume = {12},

pages = {100242},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid30792210,

title = {MicroRNA-127-3p controls murine hematopoietic stem cell maintenance by limiting differentiation},

author = {L Crisafulli and S Muggeo and P Uva and Y Wang and M Iwasaki and S Locatelli and A Anselmo and F S Colombo and C Carlo-Stella and M L Cleary and A Villa and B Gentner and F Ficara},

year  = {2019},

date = {2019-01-01},

journal = {Haematologica},

volume = {104},

number = {9},

pages = {1744--1755},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{neri_targeted_2015,

title = {Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts},

author = {Tui Neri and Sharon Muggeo and Marianna Paulis and Maria Elena Caldana and Laura Crisafulli and Dario Strina and Maria Luisa Focarelli and Francesca Faggioli and Camilla Recordati and Samantha Scaramuzza and Eugenio Scanziani and Stefano Mantero and Chiara Buracchi and Cristina Sobacchi and Angelo Lombardo and Luigi Naldini and Paolo Vezzoni and Anna Villa and Francesca Ficara},

doi = {10.1016/j.stemcr.2015.08.005},

issn = {2213-6711},

year  = {2015},

date = {2015-10-01},

journal = {Stem Cell Reports},

volume = {5},

number = {4},

pages = {558--568},

abstract = {Autosomal recessive osteopetrosis is a human bone disease mainly caused by TCIRG1 gene mutations that prevent osteoclasts resorbing activity, recapitulated by the oc/oc mouse model. Bone marrow transplantation is the only available treatment, limited by the need for a matched donor. The use of induced pluripotent stem cells (iPSCs) as an unlimited source of autologous cells to generate gene corrected osteoclasts might represent a powerful alternative. We generated iPSCs from oc/oc mice, corrected the mutation using a BAC carrying the entire Tcirg1 gene locus as a template for homologous recombination, and induced hematopoietic differentiation. Similarly to physiologic fetal hematopoiesis, iPSC-derived CD41(+) cells gradually gave rise to CD45(+) cells, which comprised both mature myeloid cells and high proliferative potential colony-forming cells. Finally, we differentiated the gene corrected iPSC-derived myeloid cells into osteoclasts with rescued bone resorbing activity. These results are promising for a future translation into the human clinical setting.},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{ficara_pbx1_2013,

title = {Pbx1 restrains myeloid maturation while preserving lymphoid potential in hematopoietic progenitors},

author = {Francesca Ficara and Laura Crisafulli and Chenwei Lin and Masayuki Iwasaki and Kevin S Smith and Luca Zammataro and Michael L Cleary},

doi = {10.1242/jcs.125435},

issn = {1477-9137},

year  = {2013},

date = {2013-07-01},

journal = {Journal of Cell Science},

volume = {126},

number = {Pt 14},

pages = {3181--3191},

abstract = {The capacity of the hematopoietic system to promptly respond to peripheral demands relies on adequate pools of progenitors able to transiently proliferate and differentiate in a regulated manner. However, little is known about factors that may restrain progenitor maturation to maintain their reservoirs. Conditional knockout mice for the Pbx1 proto-oncogene have a significant reduction in lineage-restricted progenitors in addition to a profound defect in hematopoietic stem cell (HSC) self-renewal. Through analysis of purified progenitor proliferation, differentiation capacity and transcriptional profiling, we demonstrate that Pbx1 regulates the lineage-specific output of multipotent and oligopotent progenitors. In the absence of Pbx1 multipotent progenitor (MPP) and common myeloid progenitor (CMP) pools are reduced due to aberrantly rapid myeloid maturation. This is associated with premature expression of myeloid differentiation genes and decreased maintenance of proto-oncogene transcriptional pathways, including reduced expression of Meis1, a Pbx1 dimerization partner, and its subordinate transcriptional program. Conversely, Pbx1 maintains the lymphoid differentiation potential of lymphoid-primed MPPs (LMPPs) and common lymphoid progenitors (CLPs), whose reduction in the absence of Pbx1 is associated with a defect in lymphoid priming that is also present in CMPs, which persistently express lymphoid and HSC genes underlying a previously unappreciated lineage promiscuity that is maintained by Pbx1. These results demonstrate a role for Pbx1 in restraining myeloid maturation while maintaining lymphoid potential to appropriately regulate progenitor reservoirs.},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid18462698,

title = {Pbx1 regulates self-renewal of long-term hematopoietic stem cells by maintaining their quiescence},

author = {F Ficara and M J Murphy and M Lin and M L Cleary},

year  = {2008},

date = {2008-05-01},

journal = {Cell Stem Cell},

volume = {2},

number = {5},

pages = {484--496},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid15564141,

title = {IL-3 or IL-7 increases ex vivo gene transfer efficiency in AĐA-SCIĐ BM CĐ34+ cells while maintaining in vivo lymphoid potential},

author = {F Ficara and D B Superchi and R J Hernández and C Mocchetti and N Carballido-Perrig and G Andolfi and S Deola and A Colombo and C Bordignon and J M Carballido and M G Roncarolo and A Aiuti},

year  = {2004},

date = {2004-12-01},

journal = {Mol Ther},

volume = {10},

number = {6},

pages = {1096--1108},

keywords = {},

pubstate = {published},

tppubtype = {article}

}

@article{pmid12089448c,

title = {Correction of AĐA-SCIĐ by stem cell gene therapy combined with nonmyeloablative conditioning},

year  = {2002},

date = {2002-06-01},

journal = {Science},

volume = {296},

number = {5577},

pages = {2410--2413},

keywords = {},

pubstate = {published},

tppubtype = {article}

}