Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …

Since its inception, the CRISPR/Cas9 technology has been widely utilized for the targeted
insertion of donor DNAs into mammalian genomes. A shortcoming with the earlier knock-in …

Hemoglobinopathies, such as β-thalassemia, and sickle cell disease (SCD) are caused by
abnormal structure or reduced production of β-chains and affect millions of people …

Abstract β-hemoglobinopathies are caused by abnormal or absent production of
hemoglobin in the blood due to mutations in the β-globin gene (HBB). Imbalanced …

Applying stem cell therapy in intractable diseases: a narrative review of decades of progress
and challenges - PMC Back to Top Skip to main content NIH NLM Logo Access keys NCBI …

Allogeneic hematopoietic stem cell transplantation (HSCT) is a standard therapeutic
intervention for hematological malignancies and several monogenic diseases. However, this …

Due to pioneering in vitro investigations on gene modification, gene engineering platforms
have incredibly improved to a safer and more powerful tool for the treatment of multiple …

Metachromatic leukodystrophy (MLD) is a rare genetic disorder caused by mutations in the
Arylsulfatase-A (ARSA) gene. The enzyme plays a key role in sulfatide metabolism in brain …

Chimeric antigen receptor (CAR)-modified T cells have raised among other
immunotherapies for cancer treatment, being implemented against B-cell malignancies …

Blood disorders are a group of diseases including hematological neoplasms, clotting
disorders and orphan immune deficiency diseases that affects human health. Current …