After many years of painstaking research, the potential of gene therapy to cure severe β-
thalassemia is now becoming evident through recent clinical trials. Therapeutic protocols …

Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

Hematopoietic stem cell (HSC) gene therapy has curative potential; however, its use is
limited by the morbidity and mortality associated with current chemotherapy-based …

Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen
accumulation in various tissues, including cardiac and skeletal muscles and the central …

In recent years, a growing number of clinical trials have been initiated to evaluate gene
therapy approaches for the treatment of patients with transfusion-dependent β-thalassemia …

Investigations to understand the function and control of the globin genes have led to some of
the most exciting molecular discoveries and biomedical breakthroughs of the 20th and 21st …

β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …

Gene editing is a growing gene engineering technique that allows accurate editing of a
broad spectrum of gene‐regulated diseases to achieve curative treatment and also has the …

Mesenchymal stromal cells (MSC) are promising options to cellular therapy to several
clinical disorders, mainly because of its ability to immunomodulate and differentiate into …

The clonal dynamics following hematopoietic stem progenitor cell (HSPC) transplantation
with busulfan conditioning are of great interest to the development of HSPC gene therapies …