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Items: 1 to 50 of 127

1.

Photo-activated raster scanning thermal imaging at sub-diffraction resolution.

Bouzin M, Marini M, Zeynali A, Borzenkov M, Sironi L, D'Alfonso L, Mingozzi F, Granucci F, Pallavicini P, Chirico G, Collini M.

Nat Commun. 2019 Dec 4;10(1):5523. doi: 10.1038/s41467-019-13447-0.

2.

Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment.

Laforêt P, Inoue M, Goillot E, Lefeuvre C, Cagin U, Streichenberger N, Leonard-Louis S, Brochier G, Madelaine A, Labasse C, Hedberg-Oldfors C, Krag T, Jauze L, Fabregue J, Labrune P, Milisenda J, Nadaj-Pakleza A, Sacconi S, Mingozzi F, Ronzitti G, Petit F, Schoser B, Oldfors A, Vissing J, Romero NB, Nishino I, Malfatti E.

Acta Neuropathol Commun. 2019 Oct 28;7(1):167. doi: 10.1186/s40478-019-0815-2.

3.

Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.

Le Quellec S, Dane AP, Barbon E, Bordet JC, Mingozzi F, Dargaud Y, Marais T, Biferi MG, Négrier C, Nathawani AC, Enjolras N.

Thromb Haemost. 2019 Dec;119(12):1956-1967. doi: 10.1055/s-0039-1697658. Epub 2019 Oct 28.

PMID:
31659733
4.

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle.

Poupiot J, Costa Verdera H, Hardet R, Colella P, Collaud F, Bartolo L, Davoust J, Sanatine P, Mingozzi F, Richard I, Ronzitti G.

Mol Ther Methods Clin Dev. 2019 Sep 3;15:83-100. doi: 10.1016/j.omtm.2019.08.012. eCollection 2019 Dec 13.

5.

Breaking the Barriers of Genetic and Metabolic Disorders.

Büning H, Bosch F, Mingozzi F.

Hum Gene Ther. 2019 Oct;30(10):1177-1179. doi: 10.1089/hum.2019.29093.hbu. No abstract available.

PMID:
31573872
6.

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome.

Aronson SJ, Veron P, Collaud F, Hubert A, Delahais V, Honnet G, de Knegt RJ, Junge N, Baumann U, Di Giorgio A, D'Antiga L, Ginocchio VM, Brunetti-Pierri N, Labrune P, Beuers U, Bosma PJ, Mingozzi F.

Hum Gene Ther. 2019 Oct;30(10):1297-1305. doi: 10.1089/hum.2019.143.

7.

SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson's Disease.

Espinoza S, Scarpato M, Damiani D, Managò F, Mereu M, Contestabile A, Peruzzo O, Carninci P, Santoro C, Papaleo F, Mingozzi F, Ronzitti G, Zucchelli S, Gustincich S.

Mol Ther. 2019 Aug 16. pii: S1525-0016(19)30365-X. doi: 10.1016/j.ymthe.2019.08.005. [Epub ahead of print]

8.

Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain.

Orefice NS, Souchet B, Braudeau J, Alves S, Piguet F, Collaud F, Ronzitti G, Tada S, Hantraye P, Mingozzi F, Ducongé F, Cartier N.

Mol Ther Methods Clin Dev. 2019 Jul 3;14:237-251. doi: 10.1016/j.omtm.2019.06.005. eCollection 2019 Sep 13.

9.

Progress and challenges of gene therapy for Pompe disease.

Ronzitti G, Collaud F, Laforet P, Mingozzi F.

Ann Transl Med. 2019 Jul;7(13):287. doi: 10.21037/atm.2019.04.67. Review.

10.

Gene Therapy for Pompe Disease: The Time is now.

Colella P, Mingozzi F.

Hum Gene Ther. 2019 Oct;30(10):1245-1262. doi: 10.1089/hum.2019.109. Epub 2019 Sep 9.

PMID:
31298581
11.

Whole-Section Tumor Micro-Architecture Analysis by a Two-Dimensional Phasor-Based Approach Applied to Polarization-Dependent Second Harmonic Imaging.

Scodellaro R, Bouzin M, Mingozzi F, D'Alfonso L, Granucci F, Collini M, Chirico G, Sironi L.

Front Oncol. 2019 Jun 19;9:527. doi: 10.3389/fonc.2019.00527. eCollection 2019.

12.

Balancing immunity and tolerance in gene therapy for inherited and acquired diseases.

Scott DW, Mingozzi F.

Cell Immunol. 2019 Aug;342:103945. doi: 10.1016/j.cellimm.2019.103945. Epub 2019 Jun 14. No abstract available.

PMID:
31229195
13.

Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity.

Bartolo L, Li Chung Tong S, Chappert P, Urbain D, Collaud F, Colella P, Richard I, Ronzitti G, Demengeot J, Gross DA, Mingozzi F, Davoust J.

JCI Insight. 2019 Jun 6;4(11). pii: 127008. doi: 10.1172/jci.insight.127008. eCollection 2019 Jun 6.

14.

Erratum: Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.

Han SO, Ronzitti G, Arnson B, Leborgne C, Li S, Mingozzi F, Koeberl D.

Mol Ther Methods Clin Dev. 2019 May 1;13:431. doi: 10.1016/j.omtm.2019.04.005. eCollection 2019 Jun 14.

15.

Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy.

Cabrera-Pérez R, Vila-Julià F, Hirano M, Mingozzi F, Torres-Torronteras J, Martí R.

Hum Gene Ther. 2019 Aug;30(8):985-998. doi: 10.1089/hum.2018.217. Epub 2019 Apr 24.

PMID:
30900470
16.

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.

Collaud F, Bortolussi G, Guianvarc'h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Dec 31;12:157-174. doi: 10.1016/j.omtm.2018.12.011. eCollection 2019 Mar 15.

17.

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.

Colella P, Sellier P, Costa Verdera H, Puzzo F, van Wittenberghe L, Guerchet N, Daniele N, Gjata B, Marmier S, Charles S, Simon Sola M, Ragone I, Leborgne C, Collaud F, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Nov 17;12:85-101. doi: 10.1016/j.omtm.2018.11.002. eCollection 2019 Mar 15.

18.

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans.

Kuranda K, Jean-Alphonse P, Leborgne C, Hardet R, Collaud F, Marmier S, Costa Verdera H, Ronzitti G, Veron P, Mingozzi F.

J Clin Invest. 2018 Dec 3;128(12):5267-5279. doi: 10.1172/JCI122372. Epub 2018 Oct 22.

19.

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.

Meliani A, Boisgerault F, Hardet R, Marmier S, Collaud F, Ronzitti G, Leborgne C, Costa Verdera H, Simon Sola M, Charles S, Vignaud A, van Wittenberghe L, Manni G, Christophe O, Fallarino F, Roy C, Michaud A, Ilyinskii P, Kishimoto TK, Mingozzi F.

Nat Commun. 2018 Oct 5;9(1):4098. doi: 10.1038/s41467-018-06621-3.

20.

AAV Immunogenicity: A Matter of Sensitivity.

Mingozzi F.

Mol Ther. 2018 Oct 3;26(10):2335-2336. doi: 10.1016/j.ymthe.2018.09.001. Epub 2018 Sep 18. No abstract available.

21.

Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY.

Mauhin W, Lidove O, Amelin D, Lamari F, Caillaud C, Mingozzi F, Dzangué-Tchoupou G, Arouche-Delaperche L, Douillard C, Dussol B, Leguy-Seguin V, D'Halluin P, Noel E, Zenone T, Matignon M, Maillot F, Ly KH, Besson G, Willems M, Labombarda F, Masseau A, Lavigne C, Froissart R, Lacombe D, Ziza JM, Hachulla E, Benveniste O.

Orphanet J Rare Dis. 2018 Jul 31;13(1):127. doi: 10.1186/s13023-018-0877-4.

22.

Deep Dermal Injection As a Model of Candida albicans Skin Infection for Histological Analyses.

Santus W, Mingozzi F, Vai M, Granucci F, Zanoni I.

J Vis Exp. 2018 Jun 13;(136). doi: 10.3791/57574.

23.

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15.

24.

Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model.

Donadon I, Pinotti M, Rajkowska K, Pianigiani G, Barbon E, Morini E, Motaln H, Rogelj B, Mingozzi F, Slaugenhaupt SA, Pagani F.

Hum Mol Genet. 2018 Jul 15;27(14):2466-2476. doi: 10.1093/hmg/ddy151.

25.

Author Correction: μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure.

Radaelli F, D'Alfonso L, Collini M, Mingozzi F, Marongiu L, Granucci F, Zanoni I, Chirico G, Sironi L.

Sci Rep. 2018 Apr 17;8(1):6314. doi: 10.1038/s41598-018-24800-6.

26.

Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients.

Leborgne C, Latournerie V, Boutin S, Desgue D, Quéré A, Pignot E, Collaud F, Charles S, Simon Sola M, Masat E, Jouen F, Boyer O, Masurier C, Mingozzi F, Veron P.

Cell Immunol. 2019 Aug;342:103780. doi: 10.1016/j.cellimm.2018.03.004. Epub 2018 Mar 16.

PMID:
29571923
27.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F.

Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28.

28.

Emerging Issues in AAV-Mediated In Vivo Gene Therapy.

Colella P, Ronzitti G, Mingozzi F.

Mol Ther Methods Clin Dev. 2017 Dec 1;8:87-104. doi: 10.1016/j.omtm.2017.11.007. eCollection 2018 Mar 16. Review.

29.

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors.

Meliani A, Boisgerault F, Fitzpatrick Z, Marmier S, Leborgne C, Collaud F, Simon Sola M, Charles S, Ronzitti G, Vignaud A, van Wittenberghe L, Marolleau B, Jouen F, Tan S, Boyer O, Christophe O, Brisson AR, Maguire CA, Mingozzi F.

Blood Adv. 2017 Oct 16;1(23):2019-2031. doi: 10.1182/bloodadvances.2017010181. eCollection 2017 Oct 24.

30.

Combination Therapy Is the New Gene Therapy?

Ronzitti G, Mingozzi F.

Mol Ther. 2018 Jan 3;26(1):12-14. doi: 10.1016/j.ymthe.2017.12.008. Epub 2017 Dec 20. No abstract available.

31.

μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure.

Radaelli F, D'Alfonso L, Collini M, Mingozzi F, Marongiu L, Granucci F, Zanoni I, Chirico G, Sironi L.

Sci Rep. 2017 Dec 12;7(1):17468. doi: 10.1038/s41598-017-17726-y. Erratum in: Sci Rep. 2018 Apr 17;8(1):6314.

32.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.

33.

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA.

Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25.

34.

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape.

Mingozzi F, High KA.

Annu Rev Virol. 2017 Sep 29;4(1):511-534. doi: 10.1146/annurev-virology-101416-041936. Review.

PMID:
28961410
35.

Skin infections are eliminated by cooperation of the fibrinolytic and innate immune systems.

Santus W, Barresi S, Mingozzi F, Broggi A, Orlandi I, Stamerra G, Vai M, Martorana AM, Polissi A, Köhler JR, Liu N, Zanoni I, Granucci F.

Sci Immunol. 2017 Sep 22;2(15). pii: eaan2725. doi: 10.1126/sciimmunol.aan2725.

36.

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial.

Vandamme C, Adjali O, Mingozzi F.

Hum Gene Ther. 2017 Nov;28(11):1061-1074. doi: 10.1089/hum.2017.150. Review.

37.

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G.

Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105.

38.

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.

Han SO, Ronzitti G, Arnson B, Leborgne C, Li S, Mingozzi F, Koeberl D.

Mol Ther Methods Clin Dev. 2017 Jan 11;4:126-136. doi: 10.1016/j.omtm.2016.12.010. eCollection 2017 Mar 17. Erratum in: Mol Ther Methods Clin Dev. 2019 May 01;13:431.

39.

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.

Hösel M, Huber A, Bohlen S, Lucifora J, Ronzitti G, Puzzo F, Boisgerault F, Hacker UT, Kwanten WJ, Klöting N, Blüher M, Gluschko A, Schramm M, Utermöhlen O, Bloch W, Mingozzi F, Krut O, Büning H.

Hepatology. 2017 Jul;66(1):252-265. doi: 10.1002/hep.29176. Epub 2017 May 29.

40.

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK.

Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22.

41.

Oral Tolerance: Another Reason to Eat Your Veggies!

Hardet R, Mingozzi F.

Mol Ther. 2017 Feb 1;25(2):311-313. doi: 10.1016/j.ymthe.2017.01.001. Epub 2017 Jan 18. No abstract available.

42.

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics.

Barbon E, Ferrarese M, van Wittenberghe L, Sanatine P, Ronzitti G, Collaud F, Colella P, Pinotti M, Mingozzi F.

Mol Ther Nucleic Acids. 2016 Nov 29;5(11):e392. doi: 10.1038/mtna.2016.97.

43.

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.

Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F; French Pompe Registry Study Group.

Sci Rep. 2016 Nov 4;6:36182. doi: 10.1038/srep36182.

44.

Exosome-associated AAV vector as a robust and convenient neuroscience tool.

Hudry E, Martin C, Gandhi S, György B, Scheffer DI, Mu D, Merkel SF, Mingozzi F, Fitzpatrick Z, Dimant H, Masek M, Ragan T, Tan S, Brisson AR, Ramirez SH, Hyman BT, Maguire CA.

Gene Ther. 2016 Nov;23(11):819. doi: 10.1038/gt.2016.65. No abstract available.

PMID:
27808124
45.

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F.

Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. eCollection 2016.

46.

Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products.

Martinet J, Bourdenet G, Meliani A, Jean L, Adriouch S, Cohen JL, Mingozzi F, Boyer O.

Front Immunol. 2016 Sep 15;7:360. eCollection 2016.

47.

Prolonged contact with dendritic cells turns lymph node-resident NK cells into anti-tumor effectors.

Mingozzi F, Spreafico R, Gorletta T, Cigni C, Di Gioia M, Caccia M, Sironi L, Collini M, Soncini M, Rusconi M, von Andrian UH, Chirico G, Zanoni I, Granucci F.

EMBO Mol Med. 2016 Sep 1;8(9):1039-51. doi: 10.15252/emmm.201506164. Print 2016 Sep.

48.

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.

49.

Exosome-associated AAV vector as a robust and convenient neuroscience tool.

Hudry E, Martin C, Gandhi S, György B, Scheffer DI, Mu D, Merkel SF, Mingozzi F, Fitzpatrick Z, Dimant H, Masek M, Ragan T, Tan S, Brisson AR, Ramirez SH, Hyman BT, Maguire CA.

Gene Ther. 2016 Apr;23(4):380-92. doi: 10.1038/gt.2016.11. Epub 2016 Feb 2. Erratum in: Gene Ther. 2016 Nov;23 (11):819.

50.

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.

Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E.

Mol Ther Methods Clin Dev. 2015 Sep 30;2:15029. doi: 10.1038/mtm.2015.29. eCollection 2015.

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