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Publications

 

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Year

 

 

 

 

 

Title

 

 

 

 

 

 

 

 

 

Authors

Title of periodical or of series

 

 

 

 

 

Partners

WP2

 

 

 

 

 

2014

 

 

 

 

 

Similar Therapeutic Efficacy Between a Single Administration of Gene Therapy and Multiple Administrations of Recombinant Enzyme in a Mouse Model of Lysosomal Storage Disease

 

 

 

 

 

 

 

 

 

Ferla R, Claudiani P, Cotugno G, Saccone P, De Leonibus E, Auricchio A

Human Gene Therapy

 

 

 

 

 

Partner 1

WP2

 

 

 

 

 

 

 

 

 

 

 

Combination of low-dose gene therapy and rarified enzyme replacement therapy is effective in a mouse model of lysosomal storage disease

 

 

 

 

 

 

 

 

 

Alliegro ML, Ferla R, Nusco E and Auricchio A

Submitted to EMBO Molecular Medicine

 

 

 

 

 

Partner 1

WP2

 

 

 

 

 

 

 

 

 

 

 

GLP-compliant non-clinical safety and biodistribution of a recombinant AAV2/8 vector administered intravenously for treatment of mucopolysaccharidosis type VI

 

 

 

 

 

 

 

 

 

Ferla R, Marteau JB, Pouillot S, Kozarsky K, Brown J,  Galimberti S, Valsecchi MG, Zuliani V and Auricchio A

In preparation

 

 

 

 

 

Partners

1, 2, 3, 7


WP

 

 

 

 

Year

 

 

 

 

Title

 

Authors

Title of periodical or series

 

 

 

 

Partners

WP4

 

 

 

 

2013

 

 

 

 

Macrophage Involvement in Mitral Valve Pathology in Mucopolysaccharidosis Type VI (Maroteaux–Lamy Syndrome)

 

Marion Brands, Jorine Roelants,Ronald de Krijger, Ad Bogers,Arnold Reuser,Ans van der Ploeg,and Wim Helbing

American Journal of Medical Genetics, Part A

 

 

 

 

Partner 6

WP4

 

 

 

 

2013

 

 

 

 

Mucopolysaccharidosis type VI phenotypes-genotypes and antibody response to galsulfase

 

Brands MM, Hoogeveen-Westerveld M, Kroos MA, Nobel W, Ruijter GJ, Ozkan L, Plug I, Grinberg D, Vilageliu L, Halley DJ, Ploeg AT, Reuser AJ

Orphanet Journal of Rare Diseases

 

 

 

 

Partner 6

WP4

 

 

 

 

2013

 

 

 

 

Macrophage involvement in mitral valve pathology in mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome)

 

Brands M, Roelants J, de Krijger R, Bogers A, Reuser A, van der Ploeg A, Helbing W.

American Journal of Medical Genetics, Part A

 

 

 

 

Partner 6

WP4

 

 

 

 

2013

 

 

 

 

Up to five years experience with 11 mucopolysaccharidosis type VI patients

 

Brands MM, Oussoren E, Ruijter GJ, Vollebregt AA, van den Hout HM, Joosten KF, Hop WC, Plug I, van der Ploeg AT

Molecular Genetics and Metabolism

 

 

 

 

Partner 6

WP4

 

 

 

 

2013

 

 

 

 

Mucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI

 

Brands MM, Frohn-Mulder IM, Hagemans ML, Hop WC, Oussoren E, Helbing WA, van der Ploeg AT

Journal of Inherited Metabolic Disease

 

 

 

 

Partner 6

WP4

 

 

 

 

2015

 

 

 

 

Long-term cognitive follow-up in children treated for Maroteaux-Lamy Syndrome

 

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 A

Journal of Inherited Metabolic Disease

 

 

 

 

Partner 6

WP4

 

 

 

 

2015

 

 

 

 

Prevalence of anti-AAV8 neutralizing antibodies and ARSB cross-reactive immunologic material in MPS VI patient candidates for a gene therapy trial

 

Ferla R, Claudiani P, Savarese M, Kozarsky K, 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 and Auricchio A

Human Gene Therapy

 

 

 

 

Partners

1, 2, 4, 5, 6

WP4

 

 

 

 

2015

 

 

 

 

Pain, a prevalent feature in patients with mucopolysaccharidosis. Results of a cross sectional national survey

 

Brands M.M., Gungor D, van den Hout J.M.P, Karstens F.P.J, Oussoren

Journal of Inherited Metabolic Disease

 

 

 

 

Partner 6


“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.”

 

 

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