Luigi Naldini - Selected Publications#

1. Ferrari S§, Jacob A§, Beretta S, Unali G, Albano L, Vavassori V, Cittaro D, Lazarevic D, Brombin C, Cugnata F, Kajaste-Rudnitski A, Merelli I, Genovese P, Naldini L. Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking. Nature Biotechnology 2020; 38:1298-1308. §equal contribution. doi: 10.1038/s41587-020-0551-y.

2. Schiroli G§, Conti A§, Ferrari S, Della Volpe L, Jacob A, Albano L, Beretta S, Calabria A, Vavassori V, Gasparini P, Salataj E, Ndiaye-Lobry D, Brombin C, Chaumeil J, Montini E, Merelli I, Genovese P*, Naldini L*, Di Micco R*. Precise Gene Editing Preserves Hematopoietic Stem Cell Function Following Transient p53-Mediated DNA Damage Response. Cell Stem Cell 2019; 24:551-565. §equal contribution *These Authors share senior authorship. (https://doi.org/10.1016/j.stem.2019.02.019).

3. Milani M§, Annoni A§, Moalli F, Liu T, Cesana D, Calabria A, Bartolaccini S, Biffi M, Russo F, Visigalli I, Raimondi A, Patarroyo-White S, Drager D, Cristofori P, Ayuso E, Montini E, Peters R, Iannacone M, Cantore A*, Naldini L*. Phagocytosis-Shielded Lentiviral Vectors Improve Liver Gene Therapy in Non Human Primates. Science Translational Medicine 2019; 11:493. §equal contribution *These Authors share senior authorship

4. Escobar G, Barbarossa L, Barbiera G, Norelli M, Genua M, Ranghetti A, Plati T, Camisa B, Brombin C, Cittaro D, Annoni A, Bondanza A, Ostuni R, Gentner B.* and Naldini L.*. Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens. Nature Communication 2018; 9:2896. *These Authors share senior authorship

5. Schiroli G, Ferrari S, Conway A, Jacob A, Capo V, Albano L, Plati T, Castiello MC, Sanvito F, Gennery AR, Bovolenta C, Palchaudhuri R, Scadden DT, Holmes MC, Villa A, Sitia G, Lombardo A, Genovese P, Naldini L. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. Science Translational Medicine 2017; 9: 411. (doi: 10.1126/scitranslmed.aan0820)

6. Amabile A§, Migliara A§, Capasso P, Biffi M, Cittaro D, Naldini L* and Lombardo A*. Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing. Cell 2016; 167:219-232. § equal contribution * Co-corresponding authors

7. Genovese P, Schiroli G§, Escobar G§, Di Tomaso T, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A*, Naldini L*#. Targeted genome editing in human repopulating hematopoietic stem cells. §equal contribution; *senior authorship; #corresponding author. Nature 2014; 510:235-40.

8. Biffi A.*, Montini E.*, Lorioli L., Cesani M., Fumagalli F., Plati T., Baldoli C., Martino S., Calabria A., Canale S., Benedicenti F., Vallanti G., Biasco L., Leo S., Kabbara N., Zanetti G., Rizzo W.B., Mehta N., Cicalese M.P., Casiraghi M., Boelens J.J., Del Carro U., Dow David J.D., Schmidt M., Assanelli A., Neduva V., Di Serio C., Stupka E., Gardner J., von Kalle C., Bordignon C., Ciceri F., Rovelli A., Roncarolo M.G., Aiuti A., Sessa M. and Naldini L. Therapeutic Benefit in Metachromatic Leukodystrophy by Lentiviral Hematopoietic Stem Cell Gene Therapy. Science 2013; 341(6148): 1233158. doi: 10.1126/science.

9. Aiuti A., Biasco L.§, Scaramuzza S.§, Ferrua F., Cicalese M.P., Baricordi C., Dionisio F., Calabria A., Giannelli S., Castiello M.C., Bosticardo M., Evangelio C. , Assanelli A., Casiraghi M., Di Nunzio S., Callegaro L., Benati C., Rizzardi P., Pellin D., Di Serio C., Schmidt M., Von Kalle C., Gardner J., Mehta N., Neduva V., Dow D.J., Galy A., Miniero R., Finocchi A., Metin A., Banerjee P., Orange J., Galimberti S., Valsecchi M.G., Biffi A., Montini E., Villa A., Ciceri F., Roncarolo M.G.‡ Naldini L.‡. Lentivirus-based Gene Therapy of Hematopoietic Stem Cells in Wiskott-Aldrich Syndrome. Science 2013; 341: 1233151. doi:10.1126/science.

10. Provasi E*, Genovese P *, Lombardo A, Magnani Z, Liu PQ., Reik A, Chu V, Paschon D.E., Zhang L., Kuball J., Camisa B., Bondanza A., Casorati ., Ponzoni M., Ciceri F., Bordignon C., Greenberg P.D., Holmes M.C., Gregory Philip D., Naldini L. and Bonini C. Editing T cell specificity towards leukemia by zinc-finger nucleases and lentiviral gene transfer. Nature Medicine 2012; 18:807-15. * Co-first authors: EP and PG. Corresponding authors: LN and CB.