More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared
to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The …

Integrating vectors are associated with alterations in cellular function related to disruption of
normal gene function. This has been associated with clonal expansion of cells and, in some …

Recombinant adeno-associated viral (rAAV) vectors are considered promising tools for gene
therapy directed at the liver. Whereas rAAV is thought to be an episomal vector, its single …

HIV-1 insertions targeting BACH2 or MLK2 are enriched and persist for decades in
hematopoietic cells from patients under combination antiretroviral therapy. However, it is …

Integration of new DNA into cellular genomes mediates replication of retroviruses and
transposons; integration reactions have also been adapted for use in human gene therapy …

Long-lasting immune protection from pathogens and cancer requires the generation of
memory T cells able to survive long-term. To unravel the immunological requirements for …

Analysis of sites of newly integrated DNA in cellular genomes is important to several fields,
but methods for analyzing and visualizing these datasets are still under development. Here …

Lignocellulose wastes stemming from agricultural residues can offer an excellent
opportunity as alternative energy solutions in addition to fossil fuels. Besides, the …

In order to support the clinical application of hematopoietic stem cell (HSC) gene therapy for
mucopolysaccharidosis I (MPS I), biosafety studies were conducted to assess the toxicity …

Metachromatic leukodystrophy (MLD) and globoid cell leukodystrophy (GLD or Krabbe
disease) are severe neurodegenerative lysosomal storage diseases (LSD) caused by …