Glycosaminoglycan signatures in body fluids of mucopolysaccharidosis type II mouse model under long-term enzyme replacement therapy

[11] Haddow HRM(2010)Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy Mol Cells 30 13-580

[12] Palomaki GE(1999)Targeting disruption of the mouse iduronate sulfatase gene Am J Genet 65 A427-1091

[13] Giugliani R(2006)Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery Hum Mol Genet 15 1225-464

[14] Martins AM(2017)Brain RNA-Seq profiling of the mucopolysaccharidosis type II mouse model Int J Mol Sci 18 E1072-1374

[15] Okuyama T(2005)Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts J Gene Med 7 1482-267

[16] Seo J-H(2008)Gene therapy of Hunter syndrome: evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS) Biochim Biophys Acta 1782 574-99

[17] Kosuga M(2010)Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II Br J Pharmacol 159 1082-1043

[18] Hamazaki T(2019)Iduronate-2-sulfatase with anti-human transferrin receptor antibody for neuropathic mucopolysaccharidosis II: a phase 1/2 trial Mol Ther J Am Soc Gene Ther 27 456-558

[19] Bellesso S(2019)Targeting brain disease in MPSII: preclinical evaluation of IDS-loaded PLGA nanoparticles Int J Mol Sci 20 E2014-2022

[20] Salvalaio M(2018)A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II Mol Ther J Am Soc Gene Ther 26 1366-55

[11] Haddow HRM(2010)Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy Mol Cells 30 13-580

[12] Palomaki GE(1999)Targeting disruption of the mouse iduronate sulfatase gene Am J Genet 65 A427-1091

[13] Giugliani R(2006)Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery Hum Mol Genet 15 1225-464

[14] Martins AM(2017)Brain RNA-Seq profiling of the mucopolysaccharidosis type II mouse model Int J Mol Sci 18 E1072-1374

[15] Okuyama T(2005)Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts J Gene Med 7 1482-267

[16] Seo J-H(2008)Gene therapy of Hunter syndrome: evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS) Biochim Biophys Acta 1782 574-99

[17] Kosuga M(2010)Genistein reduces glycosaminoglycan levels in a mouse model of mucopolysaccharidosis type II Br J Pharmacol 159 1082-1043

[18] Hamazaki T(2019)Iduronate-2-sulfatase with anti-human transferrin receptor antibody for neuropathic mucopolysaccharidosis II: a phase 1/2 trial Mol Ther J Am Soc Gene Ther 27 456-558

[19] Bellesso S(2019)Targeting brain disease in MPSII: preclinical evaluation of IDS-loaded PLGA nanoparticles Int J Mol Sci 20 E2014-2022

[20] Salvalaio M(2018)A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II Mol Ther J Am Soc Gene Ther 26 1366-55