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, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Ans T. van der Ploeg, Niek P. van Til, W.W.M. Pim Pijnappel
Abstract
as a candidate transgene for future clinical development of HSPC-LVGT for Pompe disease.
Topics & Concepts
Genetic enhancementBiologyTransgeneGlycogenHaematopoiesisViral vectorEnzyme replacement therapyNeurodegenerationAutophagyCancer researchStem cellEndocrinologyInternal medicineGeneMedicineDiseaseCell biologyBiochemistryRecombinant DNAApoptosisLysosomal Storage Disorders ResearchAdenosine and Purinergic SignalingGenetics and Neurodevelopmental Disorders