Publications

WP1, WP2
Ferla R, Claudiani P, Cotugno G, Saccone P, De Leonibus E, Auricchio A. Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease. Hum Gene Ther. 2014 Jul;25(7):609-18.
Alliegro M, Ferla R, Nusco E, De Leonibus C, Settembre C, Auricchio A. Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease. Mol Ther. 2016 Dec;24(12):2054-206.
Ferla R, Alliegro M, Marteau JB, Dell'Anno M, Nusco E, Pouillot S, Galimberti S, Valsecchi MG, Zuliani V, Auricchio A. Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. Mol Ther Methods Clin Dev. 2017 Jul 24;6:143-158.
WP4
Brands M, Roelants J, de Krijger R, Bogers A, Reuser A, van der Ploeg AT, Helbing W. Macrophage involvement in mitral valve pathology in mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome). Am J Med Genet A. 2013 Oct;161(10): 2550-3.
Ferla R, Claudiani P, Savarese M, Kozarsky KF, Parini R, Scarpa M, Donati MA, Sorge G, Hopwood JJ, Parenti G, Fecarotta S, Nigro V, Sivri HS, Van Der Ploeg A, Andria G, Brunetti-Pierri N, Auricchio A. Prevalence of anti-AAV8 neutralizing antibodies and ARSB cross-reactive immunologic material in MPS VI patients candidates for a gene therapy trial. Hum Gene Ther. 2015 Mar;26(3)145-52.
Brands MM, Güngör D, van den Hout H, Karstens F, Oussoren E, Plug I, Boelens JJ, van Hasselt PM, Hollak CE, Mulder MF, Gozalbo ER, Smeitink JA, Smit GP, Wijburg FA, Meutgeert H, van der Ploeg AT. Pain: a prevalent feature in patients with mucopolysaccharidosis. Results of a cross-sectional national survey. J Inherit Metab Dis. 2015 Mar;38(2):323-31.
Ebbink BJ, Brands MM, van den Hout JM, Lequin MH, van den Braak RR, van de Weitgraven RL, Plug I, Aarsen FK, van der Ploeg AT. Long-term cognitive follow-up in children treated for Maroteaux-Lamy Syndrome. J Inherit Metab Dis. 2016 Mar;39(2):285-92.
Oussoren E, Bessems JHJM, Pollet V, van der Meijden JC, van der Giessen LJ, Plug I, Devos AS, Ruijter GJG, van der Ploeg AT, Langeveld M. A long term follow-up study of the development of hip disease in Mucopolysaccharidosis type VI. Mol Genet Metab. 2017 Jul;121(3):241-251.

“The research leading to these results has received funding from the European Union’s Seventh Framework Programme [FP7/2007-2013] under grant agreement n˚ 304999.”