Naldini Luigi Professore ordinarioMedicinaBIO/17

Professore Ordinario di Istologia

Direttore Istituto San Raffaele Telethon per la Terapia Genica (SR-Tiget), Milano

Curriculum Vitae

– da Ottobre 2008: Direttore Istituto San Raffaele Telethon per la Terapia Genica (SR-Tiget), Milano

– dal 2003: Professore di I fascia di Istologia e di Terapia Genica e Cellulare, Università Vita Salute San Raffaele, Milano

– 2003-2008 Co-Direttore Istituto San Raffaele Telethon per la Terapia Genica (SR-Tiget), Milano

– 2002-1998 Professore Associato, Università di Torino e Direttore del Laboratorio di Terapia Genica, Istituto per la Ricerca e Cura del Cancro, Candiolo, Torino

– 1998-1996 Senior Scientist e Direttore del Progetto Vettori Lentivirali, Cell Genesys, Foster City, California, USA

– 1996-1994 Visiting Scientist, Laboratorio di Genetica (Direttore: Prof. Inder Verma), Salk Institute for Biological Studies, La Jolla, California, USA

– 1996-1990 Ricercatore Universitario, Dipartimento di Scienze Biomediche ed Oncologia dell’Università di Torino

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

– 1987-1983 Dottorato di Ricerca in Scienze Citologiche e Morfogenetiche con P.M. Comoglio, Università di Torino

– 1983 Laurea in Medicina e Chirurgia, Università di Torino

Attività Scientifica

Luigi Naldini è Direttore dell’Istituto San Raffaele Telethon per la Terapia Genica e Professore all’Università Vita-Salute San Raffaele di Milano. Nel corso degli ultimi 25 anni Luigi Naldini è stato pioniere nello sviluppo e nell’applicazione di vettori lentivirali per terapia genica, che sono diventati uno degli strumenti più utilizzati nella ricerca biomedica e recentemente stanno offrendo una prospettiva di cura per diverse malattie genetiche e tumorali finora inesorabilmente fatali.  La sua ricerca si é focalizzata a superare i principali ostacoli ad un sicuro ed efficace trasferimento genico, adottando soluzioni innovative che da un lato sono state tradotte in nuove strategie terapeutiche e dall’altro hanno permesso di fare nuove scoperte sulle funzioni delle cellule staminali ematopoietiche e sull’angiogenesi tumorale. Il suo lavoro ha inoltre contribuito allo sviluppo dell’editing genetico per la correzione in situ delle mutazioni e alla sua applicazione in terapia genica e cellulare. Ha pubblicato 272 articoli scientifici. SCOPUS Author h-index: 95. Negli ultimi 10 anni è stato invitato come relatore a più di 150 congressi internazionali e nazionali e come relatore Magistrale in 15 eventi.

E’ membro della società Europea di Biologia Molecolare (EMBO), è stato Presidente della Società Europea di Terapia Genica e Cellulare (ESGCT) ed è stato chiamato a far parte, in qualità di esperto, del comitato “Human Gene Editing” dell’Accademia Nazionale delle Scienze e della Medicina degli Stati Uniti d’America e del Comitato Nazionale per la Biosicurezza, le Biotecnologie e le Scienze della Vita. E’ stato insignito dell’Outstanding Achievement Award da parte della Società Americana di Terapia Genica e Cellulare (ASGCT) nel 2014 e da parte di ESGCT nel 2015, ha ricevuto il Dottorato Onorario da parte della Libera Università di Bruxelles nel 2015, il premio Jimenez Diaz nel 2016, il premio Beutler da parte della società Americana di Ematologia (ASH) nel 2017 e, nel 2019, il premio Jeantet-Collen per la Medicina Traslazionale. E’ stato nominato “Grande Ufficiale dell’Ordine al Merito della Repubblica Italiana” nel Dicembre 2019.

 

 

Pubblicazioni selezionate

 

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.

 

Annoni A, Cantore A, Della Valle P, Goudy K, Akbarpour M, Russo F, Bartolaccini S, D’Angelo A, Roncarolo MG, Naldini L. EMBO Mol Med. 2013 Nov;5(11):1684-97. Epub 2013 Sep 16.

 

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. Epub 2013 Jul 11.

* These authors contributed equally to this work.

Featured in:

Perspective: Verma I. Science 2013 Aug 23; 341(6148):853-5.

News & Views: Leboulch P. Nature 2013 Aug 15; 500(7462):280-2.

Previews: Williams D.A. Cell Stem Cell 2013 Sep 5; 263-264

 

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. Epub 2013 Jul 11.

• Equal second author contribution

‡These authors contributed equally to this work

Featured in:

Perspective: Verma I. Science 2013 Aug 23; 341(6148):853-5.

News & Views: Leboulch P. Nature 2013 Aug 15; 500(7462):280-2.

Previews: Williams D.A. Cell Stem Cell 2013 Sep 5; 263-264

 

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. Epub 2013 Mar 13.

 

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. doi: 10.1016/j.stem.2012.09.001. Epub 2012 Nov 8.

*Co-first authors: ERL, BG, PvG and AG. Co-senior corresponding authors: LN and JED.

Selected for : F1000Prime Hematology Faculty

 

Ranzani M., Cesana D.*, Bartholomä CC.*, Sanvito F., Pala M., Benedicenti F., Gallina P., Sergi Sergi L., Merella S., Bulfone A., Doglioni C., von Kalle C., KimYJ., Schmidt M., Tonon G., Naldini L. and Montini E. Lentiviral vector-based insertional mutagenesis identifies new cancer genes involved in the pathogenesis of hepatocellular carcinoma. Nature Methods. 2013 Feb;10(2):155-61. Epub 2013 Jan 13.* Equal contribution: DC and CCB. Co-senior corresponding authors: LN and EM.

 

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. Epub 2013 Feb 26.

 

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. Epub 2012 Oct 4.

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

Featured in:

Commentary: Lozier J. Blood 2012 Nov 29;120(23):4452-3.

 

Cesana D.*, Sgualdino J.*, Rudilosso L., Merella S., Naldini L. and Montini E. Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations. J Clin Invest. 2012 May 1;122(5):1667-76. Epub 2012 Apr 23.

Co-first authors: DC and JS.

Featured in:

Research Highlights: Trono D. J Clin Invest. 2012. May ;122(5):1600-2.

Research Highlights: Kreisberg J. Nature Biotechnology 2012 Jun 7;30(6):508.

 

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. Epub 2012.

* Co-first authors: EP and PG. Corresponding authors: LN and CB.

Featured in:

Research Highlights: Kreisberg J. Nature Biotechnology 2012. May 7; 30(5):411.

 

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., Bonini C. Editing T cell specificity towards leukemia by zinc-finger nucleases and lentiviral gene transfer. Nature Medicine. 2012 Apr 1. [Epub ahead of print].

*These authors equally contributed to the work. LN and CB corresponding authors.

Featured in:

Research Highlights: Kreisberg J. Nature Biotechnology. May 7; 30,411.2012

 

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). Epub 2011 Nov 1. First Authors: BDS, SMM and BG. Co-senior authors: LN and VB.

 

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.

Featured in:

Perspective: Sadelain M, Papapetrou EP, Bushman FD. Nat Rev Cancer. Dec 12:51-8.2011

 

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. Epub 2011 Aug 7. First authors: RG and AL. Co-senior and Co-corresponding authors: LN and C.von K.

Featured in:

Research Highlights: Casci T. Nature Reviews in Genetics. Aug 31;12(10):667. 2011

Community Corner: Nature Medicine. Oct 11;17(10):1192-3. 2011

News:H.Ledford. Nature News. 7 August 2011

News and Views: Mussolino C. & Cathomen T. Nature Methods. 8,725–726. 2011

 

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:5

 

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. Epub 2011 Mar 14. Co-senior authors: LN and EM.

 

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.

Featured in:

Preview: Lewis & Ferrara: Cancer Cell 2011 Apr 12;19(4):431-3

Breaking advance: Cancer Research, 2011 Apr 15

Featured Cover Article

 

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.

 

De Palma M, Naldini L. Angioprotein-2 TIEs Up Macrophages in Tumor Angiogenesis. Clin Cancer Res. 2011 May 16t. Aug 15;17(16):5226-32. Epub 2011 May 16.

 

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.

 

Mazzieri R.*, Pucci F.*, Moi D., Zonari E., Ranghetti A., Berti A., Politi LS., Gentner B., Brown JL., 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.

*: Equal Contribution. §: Co-senior & co-corresponding authors.

Naldini L. Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet. 2011 May;12(5):301-15. Epub 2011 Mar 29.

 

Biffi A., Bartolomae CC., 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 selection. Blood 2011 May 19;117(20):5332-9. Epub 2011 Mar 14. Co-senior authors: LN and EM.

 

Welford, A.F.*, Biziato, D.*, Coffelt, S.B., Nucera, S., Fisher, M., Pucci, F., Di Serio, C., Naldini L., De Palma, M.§, Tozer, G.M.§ and Lewis, C.E.§. TIE2-Expressing Macrophages Limit the Therapeutic Efficacy of the Vascular   Disrupting Agent, Combretastatin A4 Phosphate. J Clin Invest. 2011 May 2;121(5):1969-73. Epub 2011 Apr 1. *: Equal Contribution. §: Co-senior & co-corresponding authors.

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 Leukodystrophy. ScienceTranslationalMedicine 2010 Nov 17; 2(58):58ra84.

 

De Palma M, Naldini L. Antagonizing metastasis. Nature Biotechnology 2010 Apr;28(4):331-2.

 

Annoni A.,Brown B.D.,Cantore A.,Sergi Sergi L.,Naldini L.,Roncarolo M.G. In vivo Delivery of a MicroRNA Regulated Transgene Induces Antigen-specific Regulatory T Cells and Promotes Immunological Tolerance. Blood 2009 Dec 10;114(25):5152-61. Epub 2010 Sep 30.

 

Brown B.D. and Naldini L. Exploiting and antagonizing miRNA regulation for therapeutic and experimental applications. Nature Reviews Genetics 2009 Aug.;10(8):578-85.Review.

 

F Pucci, MA Venneri, D Biziato, A Nonis, D Moi, A Sica, C Di Serio, L Naldini, M. De Palma.A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes (TEMs), blood “resident” monocytes and embryonic macrophages suggests common functions and developmental relationships. Blood 2009 Jul 23; 114(4):901-14. Epub 2009 Apr 21.

FP and MAV contributed equally. Corresponding/Senior authors: LN and MDP.

 

1. Amendola,L. Passerini, F. Pucci, B. Gentner, R. Bacchetta, L. Naldini. Regulated and Multiple miRNA and siRNA Delivery into Primary Cells by a Lentiviral Platform.

Molecular Therapy 2009, Jun;17(6):1039-52. Epub 2009 Mar 17.

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. Epub 2009 Mar 23.

 

6. Gentner, G. Schira, 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. Epub 2008 Nov 30.

 

311. 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.

 

F.R. Santoni de Sio, A.Gritti, P.Cascio , M.Neri , M.Sampaolesi , C. Galli, J. Luban, L.Naldini. Lentiviral Vector Gene Transfer is limited by the proteasome at post-entry steps in various Types of Stem Cells. Stem Cells 2008 Aug;26(8):2142-52. Epub 2008 May 15.

 

1467. Brown*, B. Gentner*, A. Cantore,S. Colleoni, M.Amendola, A. Zingale, A.Baccarini, G. Lazzari, C. Galli, L.Naldini. 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)

 

306. Lombardo, P. Genovese, C. M. Beausejour, S. Colleoni, Y.-L. Lee, K. A. Kim, D. Ando, F. Urnov, C. Galli, P. D. Gregory, M. C. Holmes, L. Naldini. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nature Biotechnology 2007 Nov;25(11):1298-306. Epub 2007 Oct 28 .

 

50. De Palma, C. Murdoch, M.A. Venneri, L. Naldini, C.E. Lewis. Tie2-expressing monocytes: regulation of tumor angiogenesis and therapeutic implications. Trends Immunology 2007 Dec;28(12):545-50. Epub 2007 Nov 5.

 

C.E. Lewis, M. De Palma, Naldini L. Tie2-expressing monocytes: regulation by hypoxia and angioprotein-2. Cancer Research 2007 Sep15;67(18):8429-32.

 

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. Epub 2007,Aug.28.

 

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. Epub 2007 Feb 27.

 

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. Epub 2006 Dec 19.

 

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

 

66. De Palma and L. Naldini. Role of Haematopoietic Cells and Endothelial. Progenitors in Tumor Angiogenesis. Biochimica and Biofisica Acta-Rewies on Cancer 2006 Aug;1766(1):159-66. Epub 2006 Jun 27.

 

96. 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. Epub 2006 May 28.

 

8. Naldini. Inserting Optimism into Gene Therapy. Nature Medicine 2006 Apr;12(4):386-8.

 

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.

 

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. Epub 2006 Apr 23.

 

13. Dupré, F. Marangoni, S. Scaramuzza, S. Trifari, R. Jofra-Hernández, A. Aiuti, L. Naldini and Roncarolo M.-G.. Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA containing-lentiviral vector and non-lethal irradiation. Human Gene Therapy 2006 Mar;17(3):303-13.

 

26. 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.

 

A.Biffi and L. Naldini. Gene Therapy of Storage Disorders by Retroviral and Lentiviral Vectors. Human Gene Therapy 2005 Oct;16(10):1133-42.

 

15. 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. Epub 2004 Nov 12.

 

16. 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. Epub 2004 Dec 26.

 

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 NationalAcademy of Sciences of the USA 2004 Oct12;101(41):14835-40.

 

9. 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.

 

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.

 

206. De Palma, M.A. Venneri and L. Naldini. In vivo Targeting of Tumor Endothelial Cells by Systemic Delivery of Lentiviral Vectors. Human Gene Therapy 2003 Aug 10;14(12):1193-206.

 

95. 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. Epub 2003 May 12.

 

L.L. Ailles, M. Schmidt, F. Santoni de Sio, H. Glimm, S. Cavalieri, S. Bruno, W. Piacibello, C. Von Kalle and L. Naldini. Molecular Evidence of Lentiviral Vector Mediated Gene Transfer into Human Self-Renewing, Multi-Potent, Long-Term NOD/SCID Repopulating Hematopoietic Cells. Molecular Therapy 2002 Nov;6(5):615-26.

 

61. Vigna, S. Cavalieri, L. Ailles, M. Geuna, R. Loew, H. Bujard and L. Naldini. Robust and Efficient Regulation of Transgene Expression in Vivo by Improved Tetracycline-Dependent Lentiviral Vectors. Molecular Therapy. 2002 Mar;5(3):252-61.

 

A.Follenzi, G. Sabatino, A. Lombardo, C. Boccaccio and L. Naldini. Efficient Gene Delivery and Targeted Expression to Hepatocytes In Vivo by Improved Lentiviral Vectors. Human Gene Therapy. 2002;13: 243-260.

 

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.

 

M.A. Kay, J. Glorioso and L. Naldini. Viral Vectors for Gene Therapy: the art of turning infectious agents into vehicles of therapeutics. Nature Medicine. 2001 Jan;7(1):33-40.

 

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.

 

52. Park , K. Ohashi, W. Chiu, L. Naldini and M. A. Kay. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nature Genetics. 2000 Jan;24(1):49-52.

 

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.

 

S.S. Case, M.A. Price, C.T. Jordan, X.J. Yu, L.J. Wang, G. Bauer, D.L. Haas, D.Xu, R.Stripecke, L. Naldini, D.B. Kohn and G. M. Crooks. Stable Transduction of Quiescent CD34+CD38- Human Hematopoietic Cells by HIV-1 Based Lentiviral Vectors. Proceedings NationalAcademy of Sciences of the USA 1999 Mar 16;96(6):2988-93.

 

80. 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.

 

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.

 

5. Zufferey, 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.

 

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

 

7. Naldini, 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.