We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

Abstract β-Thalassemia and sickle cell disease (SCD) are the most prevalent monogenic
diseases. These disorders are caused by quantitative or qualitative defects in the production …

CRISPR-Cas9 is a promising technology for genome editing. Here we use Cas9 nuclease-
induced double-strand break DNA (DSB) at the UROS locus to model and correct congenital …

In higher eukaryotes, many genes are regulated by enhancers that are 104–106 base pairs
(bp) away from the promoter. Enhancers contain transcription-factor-binding sites (which are …

Abstract β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-
globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA …

Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach
for gene therapy of β-thalassemia and sickle cell disease. Using the CRISPR/Cas9 system …

The CRISPR/Cas9 system is a powerful genome editing tool that has made enormous
impacts on next-generation molecular diagnostics and therapeutics, especially for genetic …

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 …

Hydroxyurea is an antimetabolite drug that induces fetal haemoglobin in sickle cell disease.
However, its clinical usefulness in β-thalassaemia is unproven. We conducted a …

Genome editing of hematopoietic stem cells (HSCs) represents a therapeutic option for a
number of hematological genetic diseases, as HSCs have the potential for self‐renewal and …