Francesca Ficara


Area of interest:

I conducted graduate studies in hematopoietic cell biology and gene transfer at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, under the supervision of Prof. A. Aiuti and of Prof. Roncarolo. As a graduate student I established in vitro and in vivo systems for differentiation of human stem and progenitor cells into lymphocytes, work that formed the foundation for demonstrating successful gene transfer and multilineage engraftment in the treatment of ADA-SCID. After obtaining a PhD in Immunology from the University of Ancona, Italy, I conducted postdoctoral studies in the lab of Prof. Michael Cleary at Stanford University, California, US, to establish the role of the Pbx1 oncoprotein in hematopoiesis. I discovered that Pbx1 regulates self-renewal of hematopoietic stem cells by maintaining their quiescence, and that it restrains myeloid maturation while preserving lymphoid potential. Upon obtaining a Marie Curie Reintegration Grant, I then joined Humanitas Research Hospital within Dr. Vezzoni and Dr. A. Villa’s Unit, where I successfully differentiated gene-targeted iPSCs into hematopoietic cells including osteoclasts, for the treatment of ospeopetrosis. In 2011 I obtained a permanent position as Researcher with CNR, and in 2017 I became a Principal Investigator in Humanitas.

In my group we mainly study how the balance between self-renewal and differentiation is achieved and maintained in hematopoietic stem cells, since alterations of this equilibrium may lead to hematological disorders.

Most significant publications:


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

Generation of an immunodeficient mouse model of tcirg1-deficient autosomal recessive osteopetrosis Journal Article

In: Bone Rep, 12 , pp. 100242, 2020.



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

MicroRNA-127-3p controls murine hematopoietic stem cell maintenance by limiting differentiation Journal Article

In: Haematologica, 104 (9), pp. 1744–1755, 2019.



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

Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts Journal Article

In: Stem Cell Reports, 5 (4), pp. 558–568, 2015, ISSN: 2213-6711.

Abstract | Links | BibTeX


Ficara, Francesca; Crisafulli, Laura; Lin, Chenwei; Iwasaki, Masayuki; Smith, Kevin S; Zammataro, Luca; Cleary, Michael L

Pbx1 restrains myeloid maturation while preserving lymphoid potential in hematopoietic progenitors Journal Article

In: Journal of Cell Science, 126 (Pt 14), pp. 3181–3191, 2013, ISSN: 1477-9137.

Abstract | Links | BibTeX


Ficara, F; Murphy, M J; Lin, M; Cleary, M L

Pbx1 regulates self-renewal of long-term hematopoietic stem cells by maintaining their quiescence Journal Article

In: Cell Stem Cell, 2 (5), pp. 484–496, 2008.



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

IL-3 or IL-7 increases ex vivo gene transfer efficiency in AĐA-SCIĐ BM CĐ34+ cells while maintaining in vivo lymphoid potential Journal Article

In: Mol Ther, 10 (6), pp. 1096–1108, 2004.



Correction of AĐA-SCIĐ by stem cell gene therapy combined with nonmyeloablative conditioning Journal Article

In: Science, 296 (5577), pp. 2410–2413, 2002.