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 …

Abstract Severe Combined Immunodeficiency (SCID) is the most profound inborn error of
immunity affecting cellular and humoral immunity. Hematopoietic stem cell transplantation …

Recombination activating genes (RAGs) are tightly regulated during lymphoid differentiation,
and their mutations cause a spectrum of severe immunological disorders. Hematopoietic …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …

Gene therapy can be used to restore cell function in monogenic disorders or to endow cells
with new capabilities, such as improved killing of cancer cells, expression of suicide genes …

Allogeneic hematopoietic stem cell transplantation is an effective treatment to cure inborn
errors of immunity. Remarkable progress has been achieved thanks to the development and …

Patients with inborn errors of immunity (IEI) have been transplanted for more than 50 years.
Many long-term survivors have ongoing medical issues showing the need for further …

Abstract Purpose of Review The most serious DNA damage, DNA double strand breaks
(DNA-dsb), leads to mutagenesis, carcinogenesis or apoptosis if left unrepaired. Non …

Hematopoietic gene therapy is based on the transplantation of gene-modified autologous
hematopoietic stem cells and since the inception of this approach, many technological and …

Purpose The incidence of severe combined immunodeficiency (SCID) in the USA was
reported as 1 in 58,000 live births. In Arizona, it was anticipated that newborn screening …