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 …

Clinical gene therapy has faced numerous challenges and obstacles in the last few years.
However, it has made significant advances in treatment and is now in clinical trials and on …

CRISPR-Cas (clustered regularly interspaced short palindromic repeats-CRISPR-
associated) systems are adaptive immune systems that protect bacteria and archaea from …

Cancer immunotherapy has demonstrated significant efficacy in various tumors, but its
effectiveness in treating Hepatocellular Carcinoma (HCC) remains limited. Therefore, there …

Alpha‐thalassemia is an inherited blood disorder caused by impaired α‐globin chain
production, leading to anemia and other complications. Hemoglobin H (HbH) disease is …

Hematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the
treatment of neurodegenerative, metabolic disorders. The approach involves the ex vivo …

In the past three decades, human immunodeficiency virus type 1 (HIV-1)-derived vectors
were evolved and became indispensable to transduce therapeutic genes into a range of …

The β-thalassemias are a genetically heterogenous group of hereditary hematological
diseases caused by hundreds of mutations of the adult β-globin gene, leading to low or …

Eythropoiesis is the multistep process that leads to red blood cells production. This process
is dependent on hematopoietic stem cells (HSCs) and their unique capacity to self-renew …

Beta thalassemia is a hereditary hemoglobin disorder characterized by reduced or absent
synthesis of the beta globin chains of hemoglobin. It affects millions worldwide, posing …