Výsledky vyhledávání - "Villani GR, Di Napoli D"

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB

Autor: Di Domenico C, Villani GR, Di Napoli D, Nusco E, Calì G, Nitsch L, Di Natale P.

Publikováno v: American journal of medical genetics. Part A 149A (2009): 1209–1218.
info:cnr-pdr/source/autori:Di Domenico C; Villani GR, Di Napoli D; Nusco E; Calì G; Nitsch L; Di Natale P./titolo:Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB./doi:/rivista:American journal of medical genetics. Part A/anno:2009/pagina_da:1209/pagina_a:1218/intervallo_pagine:1209–1218/volume:149A

Mucopolysacccharidosis (MPS) IIIB is an inherited lysosomal storage disorder caused by the deficiency of alpha-N-acetylglucosaminidase (NAGLU). The disease is characterized by mild somatic features and severe neurological involvement with high mortal

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=cnr_________::934824d882fd9ffdb8d1d980f7281b3a
http://www.cnr.it/prodotto/i/21191

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Mucopolysaccharidoses type I gene therapy.

Autor: Hurt, Sarah C., Dickson, Patricia I., Curiel, David T.

Publikováno v: Journal of Inherited Metabolic Disease; Sep2021, Vol. 44 Issue 5, p1088-1098, 11p

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Screening for Carpal Tunnel Syndrome in Patients With Mucopolysaccharidosis.

Autor: Patel, Prajay, Antoniou, Georgia, Clark, Damian, Ketteridge, David, Williams, Nicole

Publikováno v: Journal of Child Neurology; May2020, Vol. 35 Issue 6, p410-417, 8p

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Specific Micro RNA-Regulated TetR-KRAB Transcriptional Control of Transgene Expression in Viral Vector-Transduced Cells.

Autor: Pichard, Virginie^1,2 Virginie.pichard@univ-nantes.fr, Aubert, Dominique³, Boni, Sebastien¹, Batt, Severine⁴, Ivacik, Dejana⁵, Huy Nguyen, Tuan^1,2, Arbuthnot, Patrick⁵, Ferry, Nicolas¹

Publikováno v: PLoS ONE. Dec2012, Vol. 7 Issue 12, p1-10. 10p.

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In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression.

Autor: Rahim, A A¹, Wong, A M², Ahmadi, S², Hoefer, K², Buckley, S M K³, Hughes, D A³, Nathwani, A N³, Baker, A H⁴, McVey, J H⁵, Cooper, J D², Waddington, S N¹

Publikováno v: Gene Therapy. Sep2012, Vol. 19 Issue 9, p936-946. 11p. 3 Color Photographs, 2 Black and White Photographs, 1 Diagram, 1 Chart.

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Intraperitoneal implant of recombinant encapsulated cells overexpressing alpha- l-iduronidase partially corrects visceral pathology in mucopolysaccharidosis type I mice.

Autor: Baldo, Guilherme^1,2, Mayer, Fabiana Quoos^1,3, Martinelli, Barbara¹, Meyer, Fabiola Schons⁴, Burin, Maira⁵, Meurer, Luise⁶, Tavares, Angela Maria Vicente⁷, Giugliani, Roberto^1,2,3,5, Matte, Ursula^1,3

Publikováno v: Cytotherapy (Taylor & Francis Ltd). Aug2012, Vol. 14 Issue 7, p860-867. 8p. 4 Color Photographs, 1 Chart, 2 Graphs.

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Defects in the Medial Entorhinal Cortex and Dentate Gyrus in the Mouse Model of Sanfilippo Syndrome Type B.

Autor: Ohmi, Kazuhiro¹, Zhao, Hui-Zhi¹, Neufeld, Elizabeth F.¹ eneufeld@mednet.ucla.edu

Publikováno v: PLoS ONE. 2011, Vol. 6 Issue 11, p1-10. 10p.

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Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice.

Autor: Watson, G.¹ gwatson@chori.org, Bastacky, J.¹, Belichenko, P.², Buddhikot, M.¹, Jungles, S.³, Vellard, M.³, Mobley, W. C.², Kakkis, E.³

Publikováno v: Gene Therapy. Jun2006, Vol. 13 Issue 11, p917-925. 9p. 3 Black and White Photographs, 2 Charts, 1 Graph.

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