Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Lentiviral vectors (LVs) are potent tools for the delivery of genes of interest into mammalian
cells and are now commonly utilised within the growing field of cell and gene therapy for the …

Pioneering gene therapy trials have shown that the genetic engineering of haematopoietic
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …

Despite tremendous efforts worldwide from clinicians and cancer scientists, pancreatic
ductal adenocarcinoma (PDA) remains a deadly disease for which no cure is available …

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe skin fragility disorder
caused by loss-of-function mutations in the COL7A1 gene, which encodes type VII collagen …

Abstract β-Thalassemia and sickle cell disease (SCD) are the most prevalent monogenic
diseases. These disorders are caused by quantitative or qualitative defects in the production …

Viral vector use in gene therapy has highlighted several safety concerns, including
genotoxic events. Generally, vector-mediated genotoxicity results from upregulation of …

Lentiviral vectors are the most frequently used tool to stably transfer and express genes in
the context of gene therapy for monogenic diseases. The vast majority of clinical …

Self-inactivating (SIN) lentiviral vectors (LV) have an excellent therapeutic potential as
demonstrated in preclinical studies and clinical trials. However, weaker mechanisms of …