Naldini Luigi Professore ordinarioMedicineBIO/17

Full Professor of Histology,

Director, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, Italy

Curriculum Vitae

– from October 2008: Director, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan

– from 2003: full Professor of Histology and Gene and Cell Therapy, University Vita Salute San Raffaele, Milan

– 2003-2008 Co-Director San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan

– 2002-1998 Associate Professor, University of Turin, and Director of the Gene Therapy Lab, Candiolo Cancer Institute - IRCCS, Candiolo, Turin

– 1998-1996 Senior Scientist and Director of the  Direttore del Progetto Vettori Lentivirali, Cell Genesys, Foster City, California, USA

– 1996-1994 Visiting Scientist, Laboratory of Genetics (Director: Prof. Inder Verma), Salk Institute for Biological Studies, La Jolla, California, USA

– 1996-1990 Universitary Researcher, Department of Biomedical and Oncologic Sciences of the University of Turin, Italy

– 1989-1987 Post-doctoral training with J. Schlessinger (Rockville, M.D. and King of Prussia), Maryland, USA

– 1987-1983 Ph.D. in Scienze Citologiche e Morfogenetiche with Prof. P.M. Comoglio, University of Turin  

– 1983 Medical Degree, University of Turin

Scientific Activity

Luigi Naldini is the Director of San Raffaele Telethon Institute for Gene Therapy, Milan, Italy, and Full Professor with Tenure at the San Raffaele Vita-Salute University, Milan. For the past 25 years he has pioneered the development and the applications of lentiviral vectors for gene therapy, which have become one of the most widely used tools in biomedical research and, upon recently entering clinical testing, are providing a long-sought hope of cures for several currently untreatable and otherwise deadly human diseases. Throughout this time, he has continued to investigate new strategies to overcome the major hurdles to safe and effective gene transfer, bringing about innovative solutions that not only are being translated into new therapeutic strategies for genetic disease and cancer, but have also allowed novel insights into hematopoietic stem cell function, induction of immunological tolerance, and tumor angiogenesis. His work also contributed to advance the use of artificial nucleases for targeted genome editing in cell and gene therapy. Has published 272 scientific papers. SCOPUS Author h-index: 95. During the last 10 years was invited as a Speaker in more than 150 international and national Congresses and as Keynote Speaker in more than 15 events.

Luigi received his medical degree from the University of Turin, Italy, and his PhD from the University of Rome. He is Member of the European Molecular Biology Organization (EMBO), has been President of the European Society of Gene and Cell Therapy (ESGCT), and has been appointed as an expert on the “Human Gene Editing Study” of the US National Academies of Sciences and of Medicine, and on the Italian National Committee for Biosafety, Biotechnology and Life Sciences. He was awarded the Outstanding Achievement Award from the American Society of Gene and Cell Therapy (ASGCT) in 2014 and from ESGCT in 2015, an Honorary doctorate from the Vrije University, Brussel, in 2015, the Jimenez Diaz Prize in 2016, the Beutler Prize from the American Society of Hematology (ASH) in 2017 and the 2019 Jeantet-Collen Prize for Translational Medicine. He was nominated “Grande Ufficiale dell’Ordine Al Merito della Repubblica Italiana”, one of the highest ranking honor in Italy, from the President of the Republic and the Prime Minister of Italy on December 27th 2019.

 

Selected publications

 

• 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. Nat Biotechnol. 2020 Jun 29. Online ahead of print.

Times Cited: 1

• 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 nonhuman primates. Sci Transl Med. 2019 May 22;11(493).

Times Cited: 14

• 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 Apr 4; 24(4):551-565.e8. §equal contribution *These Authors share senior authorship

Times Cited: 36

• 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* (2019)  Phagocytosis-Shielded Lentiviral Vectors Improve Liver Gene Therapy in Non Human Primates. Science Translational Medicine 2019 May 22;11(493). §equal contribution *These Authors share senior authorship

Times Cited: 14

• Ferrua F,  Cicalese M.P., Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo M.E.,  Calbi V,  Assanelli A.A., Facchini M, Fossati C, Albertazzi E, Scaramuzza S, Brigida I, Scala S, Basso-Ricci L, Pajno R, Casiraghi M,  Canarutto D, Salerio F.A,  Albert M.H., Bartoli  A., Wolf  H.M., Fiori R,  Silvani P, Gattillo S, Villa A, Biasco L,  Dott C., Culme-Seymour E.J., van Rossem K, Atkinson G, Valsecchi M.G,, Roncarolo M.G., Ciceri F, Naldini L,  Aiuti A.  Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Lancet Haematol. 2019 May;6(5):e239-e253.

Times Cited: 30

•  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, Jul 24;9(1):2896. *These Authors share senior authorship.

Times Cited: 6

• 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 Oct 11; 9(411).

Times Cited: 36

• Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products. EMBO Mol Med. 2017. 9: 737-740.

Times Cited: 79

• Milani M, Annoni A, Bartolaccini S, Biffi M, Russo F, Di Tomaso T, Raimondi A, Lengler J, Holmes MC, Scheiflinger F, Lombardo A, Cantore A, Naldini L. Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy. EMBO Mol Med. 2017 Aug 23.

Times Cited: 16

• National Academies of Sciences, Engineering, and Medicine. Human Genome Editing: Science, Ethics, and Governance (National Academies Press, Washington, DC, USA, 2017).

 

• 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 Sep 22;167(1):219-232. § equal contribution * Co-corresponding authors

Times Cited: 143

• Nucera S§, Giustacchini  A§, Boccalatte F§, Calabria A, Fanciullo C, Plati T, Ranghetti  A, Garcia-Manteiga  J,  Cittaro  D,  Benedicenti  F,  Lechman  ER,  Dick  JE,  Ponzoni  M,  Ciceri  F,  Montini  E, Gentner  B*,  Naldini  L*.  miR-126  Orchestrates  an  Oncogenic  Program  in  B-Cell  Precursor  Acute Lymphoblastic Leukemia. Cancer Cell 2016 Jun 13;29(6):905-21. § equal contribution * senior authorship.

Times Cited: 27

• Lechman  ER*,  Gentner  B*,  Ng  SW,  Schoof  EM,  van  Galen  P,  Kennedy JA,  Nucera  S,  Ciceri  F, Kaufmann  KB,  Takayama  N,  Dobson  SM,  Trotman-Grant  A,  Krivdova  G,  Elzinga  J,  Mitchell  A, Nilsson B, Hermans KG, Eppert K, Marke R, Isserlin R, Voisin V, Bader GD, Zandstra PW, Golub TR,  Ebert  BL,  Lu  J,  Minden  M,  Wang  JC,  Naldini  L,  Dick  JE.  miR-126  Regulates  Distinct  Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells. Cancer Cell. 2016 Feb 8;29(2):214-28. * Equal contribution

Times Cited: 82

• Sessa M*, Lorioli L*, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez D, Morena F, Calabria A, Fiori R, Silvani P, Rancoita MV, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, del Carro U, Natali Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. The Lancet. 2016 Jul 30;388(10043):476-87. * Equal contribution.

Times Cited: 181

• Catarinella M, Monestiroli A, Escobar G, Fiocchi A, Tran NL, Aiolfi R, Marra P, Esposito A, Cipriani F, Aldrighetti L, Iannacone M, Naldini L, Guidotti LG, Sitia G. IFNα gene/cell therapy curbs colorectal cancer colonization of the liver by acting on the hepatic microenvironment. EMBO Mol Med. 2016 Jan 14;8(2):155-70.

Times Cited: 8

 

• Bosley  KS,  Botchan  M,  Bredenoord  AL,  Carroll  D,  Charo  RA,  Charpentier  E,  Cohen  R,  Corn  J, Doudna J, Feng G, Greely HT, Isasi R, Ji W, Kim JS, Knoppers B, Lanphier E, Li J, Lovell-Badge R,  Martin  GS,  Moreno  J,  Naldini  L,  Pera  M,  Perry  AC,  Venter  JC,  Zhang  F,  Zhou  Q.  CRISPR germline engineering-the community speaks. Nat Biotechnol. 2015 May 12;33(5):478-8.

Times Cited: 80

• Cantore A., Ranzani M., Bartholomae C.C., Volpin M., Valle P.D., Sanvito F., Sergi L.S., Gallina P., Benedicenti F., Bellinger D., Raymer R., Merricks E., Bellintani F., Martin S., Doglioni C., D'Angelo A., VandenDriessche T., Chuah M.K., Schmidt M., Nichols T., Montini E., Naldini L. Liver-directed lentiviral gene therapy in a dog model of hemophilia B. Sci Transl Med. 2015 Mar 4;7(277):277ra28.

     Times Cited: 66

• 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 Jun 12;510(7504):235-40.

                                                                                                                                                 Times Cited: 323

• Río P§, Baños R§, Lombardo A§, Quintana O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri V, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia GC, Samper E,  Surralles J, Gregory PD, Holmes MC, Naldini L*, Bueren JA*. Targeted gene therapy in Fanconi Anemia patients' derived iPS cells.§ equal contribution; * senior authorship. EMBO Mol Med 2014 May 23;6(6):835-48.       

                                                                                                                                                 Times Cited: 57

• Escobar G., Ranghetti A., Ozkal-Baydin P., Squadrito M.L., Kajaste- Rudnitski A., Bondanza A., Gentner B., De Palma M., Mazzieri R. and Naldini L. Genetic Engineering of Hematopoiesis for Targeted IFN-α Delivery Inhibits Breast Cancer Progression. Science Transl Med. 2014 Jan 1;6(217):217ra3.

                                                                                                                                                Times Cited:51

• 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 Aug 23;341(6148): 1233158. * These authors contributed equally to this work.

                                                                                                                                             Times Cited:745

• 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 Aug 23; 341(6148): 1233151. § Equal second author contribution.

Times Cited:660

• Zonari E., Pucci F., Saini M., Mazzieri R., Politi LS., Gentner B., Naldini L. A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective anti-tumor responses. Blood. 2013 Jul 11;122(2):243-52.

Times Cited:62

•  Lechman ER.*, Gentner B.*, van Galen P.*, Giustacchini A.*, Saini M., Boccalatte FE., Hiramatsu H., Restuccia U., Bachi A., Voisin V., Bader GD., Dick JE. and Naldini L. Attenuation of miR-126 Activity Expands HSC In Vivo without Exhaustion. Cell Stem Cell. 2012 Dec 7;11(6):799-811. *Co-first authors: ERL, BG, PvG and AG. Co-senior corresponding authors: LN and JED.

Times Cited: 129

• Amendola M., Giustacchini A., Gentner B. and Naldini L. A Double Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector). Mol Ther. 2013 May;21(5):934-46.

Times Cited: 21

• Cantore A.*, Nair N.*, Della Valle P., Di Matteo M., Màtrai J., Sanvito F., Brombin C., Di Serio C., D’Angelo A., Chuah M., Naldini L. and Vandendriessche T. Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Blood. 2012 Nov 29;120(23):4517-20.

* Co-first authors: AC and NN. Co-senior corresponding authors: LN and TV.

• Provasi E.*, Genovese P. *, Lombardo A., Magnani Z., Liu P.Q., 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 May; 18(5):807-15. * Co-first authors: EP and PG. Corresponding authors: LN and CB.

Times Cited:272

• Di Stefano B.*, Maffioletti S.M.*, Gentner B.*, Ungaro F., Schira G., Naldini L.*, Broccoli V.*. A miRNA-Based System for Selecting and Maintaining the Pluripotent State in Human Induced Pluripotent Stem Cells. Stem Cells. 2011 Nov; 29(11). First Authors: BDS, SMM and BG. Co-senior authors: LN and VB.

Times Cited:24

• Lombardo A., Cesana D., Genovese P., Di Stefano B., Provasi E., Colombo D., Neri M., Magnani Z., Cantore A., Lo Riso P., Damo M. and Muniz Pello O., Holmes M.C., Gregory P.D., Gritti A., Broccoli V., Bonini C., Naldini L. Site-specific integration and in situ tailoring of cassette design allow “sustainable” gene transfer. Nat Methods. 2011 Aug 21;8(10):861-9.

Times Cited:218

• Gabriel R.*, Lombardo A.*, Arens A., Miller J.C., Genovese P., Kaeppel C., Nowrouzi A., Bartholomae CC., Wang J., Friedman G., Holmes M.C., Gregory P.D., Glimm H., Schmidt M., Naldini L.§ and von Kalle C.§. An unbiased genome-wide analysis of zinc finger nuclease specificity. Nat Biotechnol. 2011 Aug 7;29(9):816-23. First authors: RG and AL. Co-senior and Co-corresponding authors: LN and C. von K.

                                                                                                                                             Times Cited:376

• Mátrai J., Cantore A., Bartholomae CC., Annoni A., Wang W., Acosta-Sanchez A., Samara-Kuko E., De Waele L., Ma L., Genovese P., Damo M., Arens A., Goudy K., Nichols T.C., von Kalle C., L Chuah M.K., Roncarolo M.G., Schmidt M., Vandendriessche T., Naldini L. Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology. 2011 May;53(5):1696-707.

Times Cited:102

• Biffi A., Bartolomae C.C., Cesana D., Cartier N., Aubourg P., Ranzani M., Cesani M., Benedicenti F., Plati T., Rubagotti E., Merella S., Capotondo A., Sgualdino J., Zanetti G., von Kalle C., Schmidt M., Naldini L., Montini E. Lentiviral-vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood 2011 May 19;117(20):5332-9. Co-senior authors: LN and EM.

Times Cited:153

• Mazzieri R.*, Pucci F.*, Moi D., Zonari E., Ranghetti A., Berti A., Politi L.S., Gentner B., Brown J.L., Naldini L.§, and De Palma M.§. Targeting the Angiopoietin-2/TIE2 axis Inhibits Tumor Progression and Metastasis by Impairing Angiogenesis and Disabling Rebounds of Proangiogenic Myeloid Cells. Cancer Cell. 2011, Apr 12;19(4):512-26. First authors: RM and FP. Co-senior and Co-corresponding authors: LN and MDP.

                                                                                                                                             Times Cited:360

• Lombardo A., Cesana D., Genovese P., Di Stefano B., Provasi E., Colombo D., Neri M., Magnani Z., Cantore A., Lo Riso P., Damo M. and Muniz Pello O., Holmes MC., Gregory PD., Gritti A., Broccoli V., Bonini C., Naldini L. Site-specific integration and in situ tailoring of cassette design allow “sustainable” gene transfer. Nat Methods. 2011. Aug 21;8(10):861-9.

                                                                                                                                             TimesCited:219

• Mátrai J., Cantore A., Bartholomae CC., Annoni A., Wang W., Acosta-Sanchez A., Samara-Kuko E., De Waele L., Ma L., Genovese P., Damo M., Arens A., Goudy K., Nichols TC., von Kalle C., L Chuah MK., Roncarolo MG., Schmidt M., Vandendriessche T., Naldini L. Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology. 2011 May;53(5):1696-707.

                                                                                                                                             Times Cited:102

• Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A, Schira G, Amendola M, Quattrini A, Martino S, Orlacchio A, Dick JE, Biffi A, Naldini L. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med. 2010 Nov 17;2(58):58ra84.

Times Cited:149

• E.Montini*§, D.Cesana*, M.Schmidt, F. Sanvito,C. Bartholomae, M.Ranzani, F. Benedicenti, L.Sergi Sergi, A.Ambrosi, M.Ponzoni, C.Doglioni, C.Di Serio, C.von Kalle & L.Naldini§. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of hematopoietic stem cell gene therapy. Journal of Clinical Investigation 2009, Apr;119(4):964-75.

 Times Cited:395

• Gentner B., Schira G., A. Giustacchini, M. Amendola, B.D. Brown, M. Ponzoni and L. Naldini. Stable Knockdown of microRNA in Vivo by Engineered Lentiviral Vectors. Nature Methods 2009 Jan;6(1):63-6.

                                                                                                                                             Times Cited:256

• De Palma*§ R. Mazzieri*, L.S. Politi, F. Pucci , E. Zonari, S. Mazzoleni, G. Sitia, D. Moi, M.A. Venneri, S. Indraccolo, A. Falini, L.G. Guidotti, R. Galli, and L. Naldini§ Tumor-targeted interferon-α delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis. Cancer Cell 2008 Oct 7;14(4):299-311.

Times Cited:183

 

• Brown*B., Gentner B.*, Cantore A., ColleoniS. ,Amendola M. , Zingale A., Baccarini A., Lazzari G., Galli C., Naldini L. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nature Biotechnology 2007 Dec;25(12):1457-1467. Epub 2007 Nov 16; (*=equal contribution)

                                                                                                                                             Times Cited:404

• Lombardo A., Genovese P., Beausejour C.M., Colleoni S., Lee Y.-L., Kim K. A., Ando D., Urnov F.,. Galli C., Gregory P. D., Holmes M. C.,  Naldini L. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nature Biotechnology 2007 Nov;25(11):1298-306.

                                                                                                                                             Times Cited:672

• Brown B.D., Cantore A., Annoni A., Sergi Sergi L, Lombardo A, Della Valle P.,D’Angelo A, Naldini L. A microRNA-regulated lentiviral vector mediates stable correction of Hemophilia B mice. Blood Plenary Paper 2007 Dec 15;110(13):4144-52.

                                                                                                                                             Times Cited:218

• Venneri M.A., De Palma M., Ponzoni M., Pucci F., Scielzo C., Zonari E. , Mazzieri R., Doglioni C., Naldini L.. Identification of Proangiogenic TIE2-Expressing Monocytes (TEMs) in Human Peripheral Blood and Cancer. Blood 2007, Jun. 15;109(12):5276-85.

                                                                                                                                             Times Cited:330

• Brown BD, Sitia G, Annoni A, Hauben E, Sergi Sergi L, Zingale A, Roncarolo MG, Guidotti LG, Naldini L. In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promoters vector clearance. Blood 2007 April 1;109(7):2797-805.

                                                                                                                                             Times Cited:122

• Biffi A., Capotondo A., Fasano S., del Carro U., Marchesini S., Azuma H., Malaguti M.C., Amadio S.,  Brambilla R., Grompe M., Bordignon C., Quattrini A. and  Naldini L. Gene Therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. Journal of Clinical Investigation 2006 Nov;116 (11):3070-82.

Times Cited:148

• Montini, D. Cesana, M. Schmidt, F. Sanvito, M. Ponzoni, L. Sergi Sergi, F. Benedicenti, C. Bartholomae, A. Ambrosi, C. Di Serio, C. Doglioni, C. von Kalle, and L. Naldini. Hematopoietic Stem Cell Gene Transfer in a Tumor-Prone Mouse Model Uncovers Low Genotoxicity of Lentiviral Vector Integration. Nature Biotechnology 2006 Jun;24(6):687-96.

Times Cited:539

• F.R. Santoni de Sio, P. Cascio, A. Zingale, M. Gasparini and L. Naldini. Proteasome Activity Restricts Lentiviral Gene Transfer in Hematopoietic Stem Cells and is Down-Regulated by Cytokines that Enhance Transduction. Blood 2006 Jun 1;107(11):4257-65.

 Times Cited:59

• B.D. Brown, M.A. Venneri, A. Zingale, L. Sergi Sergi and L. Naldini. Endogenous microRNA Regulation Suppresses Transgene Expression in Hematopoietic Lineages and Enables Stable Gene Transfer. Nature Medicine 2006 May;12(5):585-91.

Times Cited:353

• De Palma, M.A. Venneri, R. Galli, L. Sergi Sergi, L.S. Politi, M. Sampaolesi and L. Naldini.Tie2 Identifies a Hematopoietic Lineage of Pro-Angogenic Monocytes Required for Tumor Vessel Formation and a Mesenchymal Population of Pericyte Progenitors. Cancer Cell 2005 Sep;8(3):211-26.

Times Cited:951

• De Palma, E. Montini, F. Santoni de Sio, A. Gentile, E. Medico, L. Naldini. Promoter Trapping Reveals Significant Differences in Integration Site Selection between MLV and HIV Vectors in Primary Hematopoietec Cells. Blood 2005 Mar 15;105(6):2307-15.

                                                                                                                                             Times Cited:145

• Amendola, M.A. Venneri, A. Biffi, E. Vigna, L. Naldini. Coordinate dual-gene transgenesis by Lentiviral Vectors Carrying Synthetic Bidirectional Promotors. Nature Biotechnology 2005 Jan;23(1):108-16.

                                                                                                                                             Times Cited:228

• A. Consiglio, A. Gritti, D. Dolcetta, A. Follenzi, C. Bordignon, F. H. Gage, A.L. Vescovi and L. Naldini. Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. Proceedings National Academy of Sciences of the USA 2004 Oct12;101(41):14835-40.

 Times Cited:143

• Follenzi, M Battaglia, A. Lombardo, A. Annoni, MG Roncarolo and L. Naldini. Targeting Lentiviral Vector Expression to Hepatocytes Limits Transgene-Specific Immune Response and Establishes Long-Term Expression of Human Antihemophilic Factor IX in Mice. Blood 2004 May 15;103(10):3700-9.

Times Cited:168

• A.Biffi, M. De Palma, A. Quattrini, U. Del Carro, S. Amadio, I. Visigalli, M. Sessa, S. Fasano, R. Brambilla, S. Marchesini, C. Bordignon and L. Naldini. Correction of Metachromatic Leukodystrophy in the Mouse Model by Transplantation of Genetically Modified Hematopoietic Stem Cells. The Journal of Clinical Investigation 2004 Apr;113(8):1118-29.

Times Cited:225

• De Palma, M.A. Venneri, C. Roca and L. Naldini. Targeting Exogenous Genes to Tumor Angiogenesis by Transplantation of Genetically modified Hematopoietic Stem Cells. Nature Medicine 2003 Jun;9(6):789-95.

Times Cited:477

• A. Consiglio, A. Quattrini, S. Martino, J.C. Bensadoun, D. Dolcetta, A. Trojani, G. Benaglia, S. Marchesini, V. Cestari, A. Oliverio, C. Bordignon and L. Naldini. In Vivo Gene Therapy of Metachromatic Leukodystrophy by Lentiviral Vectors: Correction of Neuropathology and Protection Against Learning Impairments in Affected Mice. Nature Medicine. 2001 Mar;7(3):310-6.

Times Cited:172

• A.Follenzi, L.E. Ailles, S. Bakovic, M. Geuna and L. Naldini. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nature Genetics. 2000 Jun;25(2):217-22.

Times Cited:750

• Bukovsky, J.-P. Song, and L. Naldini. Interaction of Human Immunodeficiency Virus-Derived Vectors with Wild-Type Virus in Transduced Cells. Journal of Virology 1999 Aug;73(8):7087-92.

  Times Cited:100

• Zufferey, T. Dull, R.J. Mandel, A. Bukovsky, D. Quiroz, L. Naldini and D.Trono. Self-Inactivating Lentiviral Vector For Safe And Efficient In Vivo Gene Delivery. Journal of Virology 1998 Dec;72(12):9873-80.

 Times Cited:1287

• T.Dull, R. Zufferey, M. Kelly, R.J. Mandel, M. Nguyen, D. Trono and L. Naldini. A Third-Generation Lentiviral Vector with a Conditional Packaging System. Journal of Virology 1998 Nov;72(11):8463-71.

 Times Cited:1993

• Zufferey M.  D. Nagy R. J. Mandel, L. Naldini and D. Trono. Multiply Attenuated Lentiviral Vector Achieves Efficient Gene Delivery In Vivo. Nature Biotechnology 1997 Sep;15(9):871-5.

 Times Cited:1426

• Naldini L., Blômer U., Gage F. H., Trono D. and Verma I. M. Efficient Transfer, Integration, and Sustained Long-Term Expression of the Transgene in Adult Rat Brains Injected with a Lentiviral Vector. Proceedings National Academy of Sciences of the USA 1996 Oct 15;93(21):11382-8.

 Times Cited:1181

• Naldini L. , U. Blômer, P. Gallay, D. Ory, R. Mulligan, F. H. Gage, I. M. Verma and D. Trono. In vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector. Science 1996 Apr 12;272(5259):263-7.

Times Cited:3685