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Items: 38

1.

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.

2.

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.

3.

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]

4.

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.

5.

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.

6.

Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III.

Jauze L, Monteillet L, Mithieux G, Rajas F, Ronzitti G.

Hum Gene Ther. 2019 Oct;30(10):1263-1273. doi: 10.1089/hum.2019.102. Epub 2019 Aug 27.

PMID:
31319709
7.

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.

8.

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.

9.

Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice.

Aronson SJ, Bakker RS, Shi X, Duijst S, Ten Bloemendaal L, de Waart DR, Verheij J, Ronzitti G, Oude Elferink RP, Beuers U, Paulusma CC, Bosma PJ.

J Hepatol. 2019 Jul;71(1):153-162. doi: 10.1016/j.jhep.2019.03.021. Epub 2019 Mar 29.

10.

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.

11.

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.

12.

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.

13.

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.

14.

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.

15.

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.

16.

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.

17.

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.

18.

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.

19.

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.

20.

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.

21.

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.

22.

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.

23.

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.

24.

Raft-partitioning of calcium channels regulates their function.

Ronzitti G, Bucci G, Stephens G, Chieregatti E.

Channels (Austin). 2015;9(4):169-70. doi: 10.1080/19336950.2015.1063285. No abstract available.

25.

TAAR1 Modulates Cortical Glutamate NMDA Receptor Function.

Espinoza S, Lignani G, Caffino L, Maggi S, Sukhanov I, Leo D, Mus L, Emanuele M, Ronzitti G, Harmeier A, Medrihan L, Sotnikova TD, Chieregatti E, Hoener MC, Benfenati F, Tucci V, Fumagalli F, Gainetdinov RR.

Neuropsychopharmacology. 2015 Aug;40(9):2217-27. doi: 10.1038/npp.2015.65. Epub 2015 Mar 9.

26.

Parkin regulates kainate receptors by interacting with the GluK2 subunit.

Maraschi A, Ciammola A, Folci A, Sassone F, Ronzitti G, Cappelletti G, Silani V, Sato S, Hattori N, Mazzanti M, Chieregatti E, Mulle C, Passafaro M, Sassone J.

Nat Commun. 2014 Oct 15;5:5182. doi: 10.1038/ncomms6182.

27.

GRP78 clustering at the cell surface of neurons transduces the action of exogenous alpha-synuclein.

Bellani S, Mescola A, Ronzitti G, Tsushima H, Tilve S, Canale C, Valtorta F, Chieregatti E.

Cell Death Differ. 2014 Dec;21(12):1971-83. doi: 10.1038/cdd.2014.111. Epub 2014 Aug 15.

28.

Exogenous α-synuclein decreases raft partitioning of Cav2.2 channels inducing dopamine release.

Ronzitti G, Bucci G, Emanuele M, Leo D, Sotnikova TD, Mus LV, Soubrane CH, Dallas ML, Thalhammer A, Cingolani LA, Mochida S, Gainetdinov RR, Stephens GJ, Chieregatti E.

J Neurosci. 2014 Aug 6;34(32):10603-15. doi: 10.1523/JNEUROSCI.0608-14.2014.

29.

Combined optical tweezers and laser dissector for controlled ablation of functional connections in neural networks.

Difato F, Dal Maschio M, Marconi E, Ronzitti G, Maccione A, Fellin T, Berdondini L, Chieregatti E, Benfenati F, Blau A.

J Biomed Opt. 2011 May;16(5):051306. doi: 10.1117/1.3560268.

30.

The regulation of synaptic function by alpha-synuclein.

Bellani S, Sousa VL, Ronzitti G, Valtorta F, Meldolesi J, Chieregatti E.

Commun Integr Biol. 2010 Mar;3(2):106-9.

31.

The total activity of a mixture of okadaic acid-group compounds can be calculated by those of individual analogues in a phosphoprotein phosphatase 2A assay.

Albano C, Ronzitti G, Rossini AM, Callegari F, Rossini GP.

Toxicon. 2009 May;53(6):631-7. doi: 10.1016/j.toxicon.2009.01.026.

PMID:
19673077
32.

Proteomic analysis reveals multiple patterns of response in cells exposed to a toxin mixture.

Sala GL, Ronzitti G, Sasaki M, Fuwa H, Yasumoto T, Bigiani A, Rossini GP.

Chem Res Toxicol. 2009 Jun;22(6):1077-85. doi: 10.1021/tx900044p.

PMID:
19397276
33.

Addendum to "A cytolytic assay for the measurement of palytoxin based on a cultured monolayer cell line" [Anal. Biochem. 374 (2008) 48-55].

Bellocci M, Ronzitti G, Milandri A, Melchiorre N, Grillo C, Poletti R, Yasumoto T, Rossini GP.

Anal Biochem. 2008 Oct 1;381(1):178. No abstract available.

PMID:
18927987
34.

Protein markers of algal toxin contamination in shellfish.

Ronzitti G, Milandri A, Scortichini G, Poletti R, Rossini GP.

Toxicon. 2008 Nov;52(6):705-13. doi: 10.1016/j.toxicon.2008.08.007. Epub 2008 Aug 20.

PMID:
18782583
35.

Yessotoxin induces the accumulation of altered E-cadherin dimers that are not part of adhesive structures in intact cells.

Ronzitti G, Rossini GP.

Toxicology. 2008 Feb 28;244(2-3):145-56. Epub 2007 Nov 21.

PMID:
18155345
36.

A cytolytic assay for the measurement of palytoxin based on a cultured monolayer cell line.

Bellocci M, Ronzitti G, Milandri A, Melchiorre N, Grillo C, Poletti R, Yasumoto T, Rossini GP.

Anal Biochem. 2008 Mar 1;374(1):48-55. Epub 2007 Nov 20.

PMID:
18023406
37.

Azaspiracid-1 alters the E-cadherin pool in epithelial cells.

Ronzitti G, Hess P, Rehmann N, Rossini GP.

Toxicol Sci. 2007 Feb;95(2):427-35. Epub 2006 Nov 21.

PMID:
17118960
38.

Selective disruption of the E-cadherin-catenin system by an algal toxin.

Ronzitti G, Callegari F, Malaguti C, Rossini GP.

Br J Cancer. 2004 Mar 8;90(5):1100-7.

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