Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

In the past century, the recombinant DNA technology was just an imagination that desirable
characteristics can be improved in the living bodies by controlling the expressions of target …

To date, over 1800 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional
gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute …

Background We investigated the long-term outcome of gene therapy for severe combined
immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of …

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 …

Hematopoietic stem cell (HSC) gene therapy for adenosine deaminase (ADA)–deficient
severe combined immunodeficiency (SCID) has shown limited clinical efficacy because of …

In order to enhance and target gene delivery we have previously established a novel
method, termed magnetofection, which uses magnetic force acting on gene vectors that are …

Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases
affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is …

Genetic defects in the purine salvage enzyme adenosine deaminase (ADA) lead to severe
combined immunodeficiency (SCID) with profound depletion of T, B, and natural killer cell …