Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …

Gene therapy, a medical approach that involves the correction or replacement of defective
and abnormal genes, plays an essential role in the treatment of complex and refractory …

Despite the ups and downs in the field over three decades, the science of gene therapy has
continued to advance and provide enduring treatments for increasing number of diseases …

Abstract lovo‐cel (bb1111; LentiGlobin for sickle cell disease [SCD]) gene therapy (GT)
comprises autologous transplantation of hematopoietic stem and progenitor cells …

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …

Sickle cell disease (SCD) is a hereditary blood disorder characterized by the presence of
abnormal hemoglobin molecules and thus distortion (sickling) of the red blood cells. SCD …

Abstract β-Thalassemia (BT) is one of the most common genetic diseases worldwide and is
caused by mutations affecting β-globin production. The only curative treatment is allogenic …

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 …

Sickle cell disease (SCD) is one of the most common hemoglobinopathies. Due to its high
prevalence, with about 20 million affected individuals worldwide, the development of novel …

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 …