Joon M. Pijnenburg

Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease

Erik van der Wal, Atze J. Bergsma, Joon M. Pijnenburg et al. · Molecular Therapy — Nucleic Acids · 2017 · 67 citations · Full text

Life Sciences, Genetics, Molecular Genetics +13

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

Qiushi Liang, Fabio Catalano, Eva C. Vlaar et al. · Molecular Therapy — Methods & Clinical Development · 2022 · 28 citations · Full text

Life Sciences, Biochemistry, Biotechnology +13

Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease

Qiushi Liang, Eva C. Vlaar, Fabio Catalano et al. · Molecular Therapy — Methods & Clinical Development · 2022 · 17 citations · Full text

Medicine, Genetics, Molecular Medicine +13