Domain-substituted IGF2 tag modulates targeting of lentiviral gene therapy for Hunter syndrome
Fabio Catalano, Dejan Stevic, Giacomo Zundo, Tessa F. Huizer et autres
0 citationsEMBO Molecular Medicine
Hannerieke J.M.P. van den Hout
Informations fournies par OpenAlex. Research Africa ne déduit ni nationalité, ni poste, ni coordonnées personnelles.
11Publications signalées
182Citations signalées
1Affiliations récentes
Institutions déclarées
Domaines associés
Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and MyoclonusCarbohydrate Chemistry and SynthesisNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy research
Publications récentes
A systematic review on the clinical experiences of lentiviral gene therapy in treating lysosomal diseases
Daniël A.M. Lambregts, Martha C. Faraguna, Giacomo Zundo, Isabel Gordaliza et autres
0 citationsMolecular Genetics and Metabolism
A new insight in Pompe disease: Pharmacokinetic variability after recombinant alpha-glucosidase infusion
Martha Caterina Faraguna, Edwin H. Jacobs, Marianne Hoogeveen‐Westerveld, Serena Gasperini et autres
0 citationsMolecular Genetics and Metabolism
Epilepsy is part of the central nervous system phenotype in classic infantile Pompe disease
Martha Caterina Faraguna, Alexander Broomfield, Serena Gasperini, Hernan Amartino et autres
0 citationsMolecular Genetics and Metabolism
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage
Fabio Catalano, Eva C. Vlaar, Zina Dammou, Drosos Katsavelis et autres
3 citationsHuman Gene Therapy
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Fabio Catalano, Eva C. Vlaar, Drosos Katsavelis, Zina Dammou et autres
6 citationsMolecular Therapy — Methods & Clinical Development
Broad variation in phenotypes for common GAA genotypes in Pompe disease
Monica Y. Niño, Stijn L. M. in't Groen, Douglas Oliveira Soares de Faria, Marianne Hoogeveen‐Westerveld et autres
9 citationsHuman Mutation
A Generic Assay to Detect Aberrant ARSB Splicing and mRNA Degradation for the Molecular Diagnosis of MPS VI
Mike Broeders, Kasper Smits, Busra Goynuk, Esmée Oussoren et autres
9 citationsMolecular Therapy — Methods & Clinical Development
A genetic modifier of symptom onset in Pompe disease
Atze J. Bergsma, Stijn L.M. in ‘t Groen, Jan J. A. van den Dorpel, Hannerieke J.M.P. van den Hout et autres
50 citationsEBioMedicine
Long-term neuropsychological follow-up in a patient with α-mannosidase
Johanneke B. Ebbink, Femke K. Aarsen, Esmée Oussoren, Iris Plug et autres
0 citationsMolecular Genetics and Metabolism
Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy
Léon P. F. Winkel, Joep H. J. Kamphoven, Hannerieke J.M.P. van den Hout, Lies Anne Severijnen et autres
105 citationsMuscle & Nerve
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