Lentiviral vectors (LVs) have provided an efficient way to integrate our gene of interest into
eukaryote cells. Human immunodeficiency virus (HIV)-derived LVs have been vastly studied …

The low gene manipulation efficiency of human hematopoietic stem and progenitor cells
(HSPC) remains a major hurdle for sustainable and broad clinical application of innovative …

Metachromatic leukodystrophy, a fatal pediatric neurodegenerative lysosomal storage
disease caused by mutations in the arylsulfatase A (ARSA) gene, is characterized by …

Abstract Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disease (LSD)
caused by a deficiency of the iduronate-2-sulfatase (IDS) that catabolizes …

Genetic defects in recombination activating genes (RAG) 1 and 2 cause a broad spectrum of
severe immune defects ranging from early severe and repeated infections to inflammation …

GM1-gangliosidosis is a progressive neurodegenerative glycosphingolipidosis resulting
from a GLB1 gene mutation causing a deficiency of the lysosomal enzyme β-galactosidase …

The work described in this thesis focuses on the study of vector-host interactions in the
context of human hematopoietic stem cell gene therapy. We study the innate immune …

Transfection, the process of introducing purified nucleic acids into cells, and viral
transduction, viral-mediated nucleic acid transfer, are two commonly utilized techniques for …

Abstract “Gene therapy” is the introduction, removal, or alteration of a person's genetic code
in order to treat or prevent disease. Many different sub-types of gene therapy exist. Some …

Endothelial progenitor cells (EPCs), a subset of bone-marrow derived pluripotent
mononuclear cells, have been touted for their prognostic, diagnostic, and therapeutic value …