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Items: 1 to 50 of 252

1.

Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells.

Petrillo C, Thorne LG, Unali G, Schiroli G, Giordano AMS, Piras F, Cuccovillo I, Petit SJ, Ahsan F, Noursadeghi M, Clare S, Genovese P, Gentner B, Naldini L, Towers GJ, Kajaste-Rudnitski A.

Cell Stem Cell. 2018 Dec 6;23(6):820-832.e9. doi: 10.1016/j.stem.2018.10.008. Epub 2018 Nov 8.

2.

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia.

Lidonnici MR, Paleari Y, Tiboni F, Mandelli G, Rossi C, Vezzoli M, Aprile A, Lederer CW, Ambrosi A, Chanut F, Sanvito F, Calabria A, Poletti V, Mavilio F, Montini E, Naldini L, Cristofori P, Ferrari G.

Mol Ther Methods Clin Dev. 2018 Sep 13;11:9-28. doi: 10.1016/j.omtm.2018.09.001. eCollection 2018 Dec 14.

3.

Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens.

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, Naldini L.

Nat Commun. 2018 Jul 24;9(1):2896. doi: 10.1038/s41467-018-05315-0.

4.

Modulation of immune responses in lentiviral vector-mediated gene transfer.

Annoni A, Gregori S, Naldini L, Cantore A.

Cell Immunol. 2018 Apr 27. pii: S0008-8749(18)30187-4. doi: 10.1016/j.cellimm.2018.04.012. [Epub ahead of print] Review.

5.

Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.

Calbi V, Fumagalli F, Consiglieri G, Penati R, Acquati S, Redaelli D, Attanasio V, Marcella F, Cicalese MP, Migliavacca M, Barzaghi F, Ferrua F, Assanelli A, Silvani P, Zoccolillo M, Chidini G, Chiesa R, Arora R, Ciotti F, Sarzana M, Antonioli G, Baldoli C, Morena F, Martino S, Ardissino GL, Sora MGN, Naldini L, Ciceri F, Aiuti A, Bernardo ME.

Bone Marrow Transplant. 2018 Jul;53(7):913-917. doi: 10.1038/s41409-017-0085-1. Epub 2018 Jan 29. No abstract available.

6.

Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next-generation human artificial chromosomes for Duchenne muscular dystrophy.

Benedetti S, Uno N, Hoshiya H, Ragazzi M, Ferrari G, Kazuki Y, Moyle LA, Tonlorenzi R, Lombardo A, Chaouch S, Mouly V, Moore M, Popplewell L, Kazuki K, Katoh M, Naldini L, Dickson G, Messina G, Oshimura M, Cossu G, Tedesco FS.

EMBO Mol Med. 2018 Feb;10(2):254-275. doi: 10.15252/emmm.201607284.

7.

Ectopic FOXP3 Expression Preserves Primitive Features Of Human Hematopoietic Stem Cells While Impairing Functional T Cell Differentiation.

Santoni de Sio FR, Passerini L, Valente MM, Russo F, Naldini L, Roncarolo MG, Bacchetta R.

Sci Rep. 2017 Nov 17;7(1):15820. doi: 10.1038/s41598-017-15689-8.

8.

The Renaissance of Gene and Cell Therapy: Florence 2016.

Naldini L, Parker GC, Flotte TR.

Hum Gene Ther. 2016 Oct;27(10):727-728. doi: 10.1089/hum.2016.29034.lna. No abstract available.

PMID:
29112477
9.

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.

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.

Sci Transl Med. 2017 Oct 11;9(411). pii: eaan0820. doi: 10.1126/scitranslmed.aan0820.

PMID:
29021165
10.

Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients.

Diez B, Genovese P, Roman-Rodriguez FJ, Alvarez L, Schiroli G, Ugalde L, Rodriguez-Perales S, Sevilla J, Diaz de Heredia C, Holmes MC, Lombardo A, Naldini L, Bueren JA, Rio P.

EMBO Mol Med. 2017 Nov;9(11):1574-1588. doi: 10.15252/emmm.201707540.

11.

Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy.

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.

EMBO Mol Med. 2017 Nov;9(11):1558-1573. doi: 10.15252/emmm.201708148.

12.

Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells.

Piras F, Riba M, Petrillo C, Lazarevic D, Cuccovillo I, Bartolaccini S, Stupka E, Gentner B, Cittaro D, Naldini L, Kajaste-Rudnitski A.

EMBO Mol Med. 2017 Sep;9(9):1198-1211. doi: 10.15252/emmm.201707922.

13.

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease.

Mastaglio S, Genovese P, Magnani Z, Ruggiero E, Landoni E, Camisa B, Schiroli G, Provasi E, Lombardo A, Reik A, Cieri N, Rocchi M, Oliveira G, Escobar G, Casucci M, Gentner B, Spinelli A, Mondino A, Bondanza A, Vago L, Ponzoni M, Ciceri F, Holmes MC, Naldini L, Bonini C.

Blood. 2017 Aug 3;130(5):606-618. doi: 10.1182/blood-2016-08-732636. Epub 2017 Jun 21.

14.

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.

Aiuti A, Roncarolo MG, Naldini L.

EMBO Mol Med. 2017 Jun;9(6):737-740. doi: 10.15252/emmm.201707573. No abstract available.

15.

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy.

Zonari E, Desantis G, Petrillo C, Boccalatte FE, Lidonnici MR, Kajaste-Rudnitski A, Aiuti A, Ferrari G, Naldini L, Gentner B.

Stem Cell Reports. 2017 Apr 11;8(4):977-990. doi: 10.1016/j.stemcr.2017.02.010. Epub 2017 Mar 16.

16.

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing.

Amabile A, Migliara A, Capasso P, Biffi M, Cittaro D, Naldini L, Lombardo A.

Cell. 2016 Sep 22;167(1):219-232.e14. doi: 10.1016/j.cell.2016.09.006.

17.

Lentiviral vectors, two decades later.

Naldini L, Trono D, Verma IM.

Science. 2016 Sep 9;353(6304):1101-2. doi: 10.1126/science.aah6192. No abstract available.

PMID:
27609877
18.

Debate on Germline Gene Editing.

Cartier-Lacave N, Ali R, Ylä-Herttuala S, Kato K, Baetschi B, Lovell-Badge R, Naldini L, Thrasher A.

Hum Gene Ther Methods. 2016 Aug;27(4):135-42. doi: 10.1089/hgtb.2016.28999.deb. No abstract available.

PMID:
27529507
19.

Leukocytes recruited by tumor-derived HMGB1 sustain peritoneal carcinomatosis.

Cottone L, Capobianco A, Gualteroni C, Monno A, Raccagni I, Valtorta S, Canu T, Di Tomaso T, Lombardo A, Esposito A, Moresco RM, Maschio AD, Naldini L, Rovere-Querini P, Bianchi ME, Manfredi AA.

Oncoimmunology. 2016 Jan 8;5(5):e1122860. doi: 10.1080/2162402X.2015.1122860. eCollection 2016 May.

20.

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies.

Visigalli I, Delai S, Ferro F, Cecere F, Vezzoli M, Sanvito F, Chanut F, Benedicenti F, Spinozzi G, Wynn R, Calabria A, Naldini L, Montini E, Cristofori P, Biffi A.

Hum Gene Ther. 2016 Oct;27(10):813-829. doi: 10.1089/hum.2016.068.

PMID:
27431943
21.

Safer conditioning for blood stem cell transplants.

Aiuti A, Naldini L.

Nat Biotechnol. 2016 Jul 12;34(7):721-3. doi: 10.1038/nbt.3629. No abstract available.

PMID:
27404882
22.

miRNA-126 Orchestrates an Oncogenic Program in B Cell Precursor Acute Lymphoblastic Leukemia.

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.

Cancer Cell. 2016 Jun 13;29(6):905-921. doi: 10.1016/j.ccell.2016.05.007.

23.

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.

Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, Canale S, Lopez ID, Morena F, Calabria A, Fiori R, Silvani P, Rancoita PM, Gabaldo M, Benedicenti F, Antonioli G, Assanelli A, Cicalese MP, Del Carro U, Sora MG, Martino S, Quattrini A, Montini E, Di Serio C, Ciceri F, Roncarolo MG, Aiuti A, Naldini L, Biffi A.

Lancet. 2016 Jul 30;388(10043):476-87. doi: 10.1016/S0140-6736(16)30374-9. Epub 2016 Jun 8.

PMID:
27289174
24.

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases.

Biasco L, Pellin D, Scala S, Dionisio F, Basso-Ricci L, Leonardelli L, Scaramuzza S, Baricordi C, Ferrua F, Cicalese MP, Giannelli S, Neduva V, Dow DJ, Schmidt M, Von Kalle C, Roncarolo MG, Ciceri F, Vicard P, Wit E, Di Serio C, Naldini L, Aiuti A.

Cell Stem Cell. 2016 Jul 7;19(1):107-19. doi: 10.1016/j.stem.2016.04.016. Epub 2016 May 26.

25.

Angiopoietin 2 expression in the cornea and its control of corneal neovascularisation.

Ferrari G, Giacomini C, Bignami F, Moi D, Ranghetti A, Doglioni C, Naldini L, Rama P, Mazzieri R.

Br J Ophthalmol. 2016 Jul;100(7):1005-1010. doi: 10.1136/bjophthalmol-2015-307901. Epub 2016 May 4.

PMID:
27146154
26.

miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells.

Lechman ER, Gentner B, Ng SWK, 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 JCY, Naldini L, Dick JE.

Cancer Cell. 2016 Apr 11;29(4):602-606. doi: 10.1016/j.ccell.2016.03.015. No abstract available.

27.

Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.

Meneghini V, Lattanzi A, Tiradani L, Bravo G, Morena F, Sanvito F, Calabria A, Bringas J, Fisher-Perkins JM, Dufour JP, Baker KC, Doglioni C, Montini E, Bunnell BA, Bankiewicz K, Martino S, Naldini L, Gritti A.

EMBO Mol Med. 2016 May 2;8(5):489-510. doi: 10.15252/emmm.201505850. Print 2016 May.

28.

miR-126 Regulates Distinct Self-Renewal Outcomes in Normal and Malignant Hematopoietic Stem Cells.

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.

Cancer Cell. 2016 Feb 8;29(2):214-28. doi: 10.1016/j.ccell.2015.12.011. Epub 2016 Jan 28. Erratum in: Cancer Cell. 2016 Apr 11;29(4):602-6.

29.

IFNα gene/cell therapy curbs colorectal cancer colonization of the liver by acting on the hepatic microenvironment.

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.

EMBO Mol Med. 2016 Feb 1;8(2):155-70. doi: 10.15252/emmm.201505395.

30.

Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy.

Ungari S, Montepeloso A, Morena F, Cocchiarella F, Recchia A, Martino S, Gentner B, Naldini L, Biffi A.

Mol Ther Methods Clin Dev. 2015 Oct 14;2:15038. doi: 10.1038/mtm.2015.38. eCollection 2015.

31.

Gene therapy returns to centre stage.

Naldini L.

Nature. 2015 Oct 15;526(7573):351-60. doi: 10.1038/nature15818. Review.

PMID:
26469046
32.

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

Neri T, Muggeo S, Paulis M, Caldana ME, Crisafulli L, Strina D, Focarelli ML, Faggioli F, Recordati C, Scaramuzza S, Scanziani E, Mantero S, Buracchi C, Sobacchi C, Lombardo A, Naldini L, Vezzoni P, Villa A, Ficara F.

Stem Cell Reports. 2015 Oct 13;5(4):558-68. doi: 10.1016/j.stemcr.2015.08.005. Epub 2015 Sep 3.

33.

MicroRNA-223 dose levels fine tune proliferation and differentiation in human cord blood progenitors and acute myeloid leukemia.

Gentner B, Pochert N, Rouhi A, Boccalatte F, Plati T, Berg T, Sun SM, Mah SM, Mirkovic-Hösle M, Ruschmann J, Muranyi A, Leierseder S, Argiropoulos B, Starczynowski DT, Karsan A, Heuser M, Hogge D, Camargo FD, Engelhardt S, Döhner H, Buske C, Jongen-Lavrencic M, Naldini L, Humphries RK, Kuchenbauer F.

Exp Hematol. 2015 Oct;43(10):858-868.e7. doi: 10.1016/j.exphem.2015.05.018. Epub 2015 Jul 8.

34.

Insulin B chain 9-23 gene transfer to hepatocytes protects from type 1 diabetes by inducing Ag-specific FoxP3+ Tregs.

Akbarpour M, Goudy KS, Cantore A, Russo F, Sanvito F, Naldini L, Annoni A, Roncarolo MG.

Sci Transl Med. 2015 May 27;7(289):289ra81. doi: 10.1126/scitranslmed.aaa3032.

PMID:
26019217
35.

CRISPR germline engineering--the community speaks.

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.

Nat Biotechnol. 2015 May;33(5):478-86. doi: 10.1038/nbt.3227. No abstract available.

PMID:
25965754
36.

Fighting rare diseases: the model of the telethon research institutes in Italy.

Ballabio A, Naldini L.

Hum Gene Ther. 2015 Apr;26(4):183-5. doi: 10.1089/hum.2015.2501. No abstract available.

PMID:
25901396
37.

Cellular innate immunity and restriction of viral infection: implications for lentiviral gene therapy in human hematopoietic cells.

Kajaste-Rudnitski A, Naldini L.

Hum Gene Ther. 2015 Apr;26(4):201-9. doi: 10.1089/hum.2015.036. Epub 2015 Apr 13. Review.

PMID:
25808164
38.

B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome.

Castiello MC, Scaramuzza S, Pala F, Ferrua F, Uva P, Brigida I, Sereni L, van der Burg M, Ottaviano G, Albert MH, Grazia Roncarolo M, Naldini L, Aiuti A, Villa A, Bosticardo M.

J Allergy Clin Immunol. 2015 Sep;136(3):692-702.e2. doi: 10.1016/j.jaci.2015.01.035. Epub 2015 Mar 16.

39.

Shedding of clinical-grade lentiviral vectors is not detected in a gene therapy setting.

Cesani M, Plati T, Lorioli L, Benedicenti F, Redaelli D, Dionisio F, Biasco L, Montini E, Naldini L, Biffi A.

Gene Ther. 2015 Jun;22(6):496-502. doi: 10.1038/gt.2015.10. Epub 2015 Mar 12.

PMID:
25762283
40.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, D'Angelo A, VandenDriessche T, Chuah MK, Schmidt M, Nichols T, Montini E, Naldini L.

Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405.

41.

The impact of amino acid variability on alloreactivity defines a functional distance predictive of permissive HLA-DPB1 mismatches in hematopoietic stem cell transplantation.

Crivello P, Zito L, Sizzano F, Zino E, Maiers M, Mulder A, Toffalori C, Naldini L, Ciceri F, Vago L, Fleischhauer K.

Biol Blood Marrow Transplant. 2015 Feb;21(2):233-41. doi: 10.1016/j.bbmt.2014.10.017. Epub 2014 Oct 23.

42.

Genome editing: a tool for research and therapy: targeted genome editing hits the clinic.

Lombardo A, Naldini L.

Nat Med. 2014 Oct;20(10):1101-3. doi: 10.1038/nm.3721. No abstract available.

PMID:
25295939
43.

Cyclosporin a and rapamycin relieve distinct lentiviral restriction blocks in hematopoietic stem and progenitor cells.

Petrillo C, Cesana D, Piras F, Bartolaccini S, Naldini L, Montini E, Kajaste-Rudnitski A.

Mol Ther. 2015 Feb;23(2):352-62. doi: 10.1038/mt.2014.193. Epub 2014 Oct 1.

44.

Lentiviral vector-based insertional mutagenesis identifies genes involved in the resistance to targeted anticancer therapies.

Ranzani M, Annunziato S, Calabria A, Brasca S, Benedicenti F, Gallina P, Naldini L, Montini E.

Mol Ther. 2014 Dec;22(12):2056-68. doi: 10.1038/mt.2014.174. Epub 2014 Sep 8.

45.
46.

Charting a clear path: the ASGCT Standardized Pathways Conference.

Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR.

Mol Ther. 2014 Jul;22(7):1235-1238. doi: 10.1038/mt.2014.95. No abstract available.

47.

Loss of transcriptional control over endogenous retroelements during reprogramming to pluripotency.

Friedli M, Turelli P, Kapopoulou A, Rauwel B, Castro-Díaz N, Rowe HM, Ecco G, Unzu C, Planet E, Lombardo A, Mangeat B, Wildhaber BE, Naldini L, Trono D.

Genome Res. 2014 Aug;24(8):1251-9. doi: 10.1101/gr.172809.114. Epub 2014 May 30.

48.

Targeted genome editing in human repopulating haematopoietic stem cells.

Genovese P, Schiroli G, Escobar G, Tomaso TD, 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.

Nature. 2014 Jun 12;510(7504):235-240. doi: 10.1038/nature13420. Epub 2014 May 28.

49.

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

Chiriaco M, Farinelli G, Capo V, Zonari E, Scaramuzza S, Di Matteo G, Sergi LS, Migliavacca M, Hernandez RJ, Bombelli F, Giorda E, Kajaste-Rudnitski A, Trono D, Grez M, Rossi P, Finocchi A, Naldini L, Gentner B, Aiuti A.

Mol Ther. 2014 Aug;22(8):1472-1483. doi: 10.1038/mt.2014.87. Epub 2014 May 29.

50.

Targeted gene therapy and cell reprogramming in Fanconi anemia.

Rio P, Baños R, Lombardo A, Quintana-Bustamante O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, Samper E, Surralles J, Gregory PD, Holmes MC, Naldini L, Bueren JA.

EMBO Mol Med. 2014 Jun;6(6):835-48. doi: 10.15252/emmm.201303374. Epub 2014 Apr 6.

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