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Items: 30


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.


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.


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.


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.


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.


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.


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.


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.


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.


Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.

Trapani I, Toriello E, de Simone S, Colella P, Iodice C, Polishchuk EV, Sommella A, Colecchi L, Rossi S, Simonelli F, Giunti M, Bacci ML, Polishchuk RS, Auricchio A.

Hum Mol Genet. 2015 Dec 1;24(23):6811-25. doi: 10.1093/hmg/ddv386. Epub 2015 Sep 29.


Hippocampus is place of interaction between unconscious and conscious memories.

Züst MA, Colella P, Reber TP, Vuilleumier P, Hauf M, Ruch S, Henke K.

PLoS One. 2015 Mar 31;10(3):e0122459. doi: 10.1371/journal.pone.0122459. eCollection 2015.


Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.

Colella P, Trapani I, Cesi G, Sommella A, Manfredi A, Puppo A, Iodice C, Rossi S, Simonelli F, Giunti M, Bacci ML, Auricchio A.

Gene Ther. 2014 Apr;21(4):450-6. doi: 10.1038/gt.2014.8. Epub 2014 Feb 27.


Effective delivery of large genes to the retina by dual AAV vectors.

Trapani I, Colella P, Sommella A, Iodice C, Cesi G, de Simone S, Marrocco E, Rossi S, Giunti M, Palfi A, Farrar GJ, Polishchuk R, Auricchio A.

EMBO Mol Med. 2014 Feb;6(2):194-211. doi: 10.1002/emmm.201302948. Epub 2013 Dec 15.


Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.

Colella P, Sommella A, Marrocco E, Di Vicino U, Polishchuk E, Garcia Garrido M, Seeliger MW, Polishchuk R, Auricchio A.

PLoS One. 2013 Aug 26;8(8):e72027. doi: 10.1371/journal.pone.0072027. eCollection 2013.


Photoreceptor degeneration in mice: adeno-associated viral vector-mediated delivery of erythropoietin.

Colella P, Auricchio A.

Methods Mol Biol. 2013;982:237-63. doi: 10.1007/978-1-62703-308-4_16.


Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.

Colella P, Auricchio A.

Hum Gene Ther. 2012 Aug;23(8):796-807. doi: 10.1089/hum.2012.123. Review.


TFEB links autophagy to lysosomal biogenesis.

Settembre C, Di Malta C, Polito VA, Garcia Arencibia M, Vetrini F, Erdin S, Erdin SU, Huynh T, Medina D, Colella P, Sardiello M, Rubinsztein DC, Ballabio A.

Science. 2011 Jun 17;332(6036):1429-33. doi: 10.1126/science.1204592. Epub 2011 May 26.


Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration.

Colella P, Iodice C, Di Vicino U, Annunziata I, Surace EM, Auricchio A.

Hum Mol Genet. 2011 Jun 1;20(11):2251-62. doi: 10.1093/hmg/ddr115. Epub 2011 Mar 19.


AAV-mediated gene supply for treatment of degenerative and neovascular retinal diseases.

Colella P, Auricchio A.

Curr Gene Ther. 2010 Oct;10(5):371-80. Review.


An all-speed projection and filtering method for gravity-stratified flows.

Gatti-Bono C, Colella P.

Philos Trans A Math Phys Eng Sci. 2009 Nov 28;367(1907):4543-58. doi: 10.1098/rsta.2009.0169.


Ocular gene therapy: current progress and future prospects.

Colella P, Cotugno G, Auricchio A.

Trends Mol Med. 2009 Jan;15(1):23-31. doi: 10.1016/j.molmed.2008.11.003. Epub 2008 Dec 25. Review.


Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

Allocca M, Doria M, Petrillo M, Colella P, Garcia-Hoyos M, Gibbs D, Kim SR, Maguire A, Rex TS, Di Vicino U, Cutillo L, Sparrow JR, Williams DS, Bennett J, Auricchio A.

J Clin Invest. 2008 May;118(5):1955-64. doi: 10.1172/JCI34316.


AP20187-mediated activation of a chimeric insulin receptor results in insulin-like actions in skeletal muscle and liver of diabetic mice.

Cotugno G, Formisano P, Giacco F, Colella P, Beguinot F, Auricchio A.

Hum Gene Ther. 2007 Feb;18(2):106-17.


Numerical computation of diffusion on a surface.

Schwartz P, Adalsteinsson D, Colella P, Arkin AP, Onsum M.

Proc Natl Acad Sci U S A. 2005 Aug 9;102(32):11151-6. Epub 2005 Aug 2.


Be careful about drawing conclusions.

Ward-Smith R, Palmer D, Colella P, Graham M, Miklea J, Mueller J, Wahba O, Guenter D.

Can Fam Physician. 2000 Sep;46:1734, 1737. No abstract available.


Numerical simulation of incompressible viscous flow in deforming domains.

Colella P, Trebotich DP.

Proc Natl Acad Sci U S A. 1999 May 11;96(10):5378-81.


Treatment of obese patients with obstructive sleep apnea syndrome (OSAS): effect of weight loss and interference of otorhinolaryngoiatric pathology.

Pasquali R, Colella P, Cirignotta F, Mondini S, Gerardi R, Buratti P, Rinaldi Ceroni A, Tartari F, Schiavina M, Melchionda N, et al.

Int J Obes. 1990 Mar;14(3):207-17.


Benfluorex action on metabolic control and insulin sensitivity in type 2 non-insulin dependent diabetics.

Pasquali R, Colella P, Capelli M, Zannarini L, Melchionda N, Barbara L.

Panminerva Med. 1989 Jul-Sep;31(3):114-8.


Body fat distribution and weight loss in obese women.

Pasquali R, Casimirri F, Colella P, Melchionda N.

Am J Clin Nutr. 1989 Jan;49(1):185-7. No abstract available.


Diabetic autonomic neuropathy and impaired human pancreatic polypeptide secretion in response to food.

Lugari R, Gnudi A, Dall'Argine P, Vattini A, Rignanese G, Dall'Aglio B, Melchionda N, Colella P, Luciani A, Toscani S, et al.

J Clin Endocrinol Metab. 1987 Feb;64(2):279-82.


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