Screening of Chimeric GAA Variants in a Preclinical Study of Pompe Disease Results in Candidate Vectors for Hematopoietic Stem Cell Gene Therapy

Lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy was investigated in a Pompe mouse model using a clinically relevant promoter driving nine engineered acid alpha-glucosidase (GAA) coding sequences incorporating distinct peptide tags and codon-optimizations.