Chimeric Enzymes Enhance Treatment Potential for Globoid Cell Leukodystrophy through Hematopoietic Stem Cell Gene Therapy

The authors presented a strategy using lentiviral vector-mediated ex vivo hematopoietic stem/progenitor cell transplantation-gene therapy with a chimeric galactosylceramidase enzyme that incorporates peptides from alpha-L-iduronidase and apolipoprotein E II to enhance expression and blood-brain barrier penetration.