Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …

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 …

Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …

Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …

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 …

β-Thalassemias Defective synthesis of the β-globin chain causes recessively inherited
disorders characterized by inadequate hemoglobin production and chronic anemia …

Background Severe combined immunodeficiency due to adenosine deaminase (ADA)
deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. Methods We …

Despite many reports of putative stem-cell-based treatments in genetic and degenerative
disorders or severe injuries, the number of proven stem cell therapies has remained small …

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 …

Sickle cell disease (SCD) is caused by a single amino acid change in the adult hemoglobin
(Hb) β chain that causes Hb polymerization and red blood cell (RBC) sickling. The co …