Since the advent of induced pluripotent stem cell (iPSC) technology a decade ago,
enormous progress has been made in stem cell biology and regenerative medicine. Human …

The CRISPR system is a revolutionary genome editing tool that has the potential to
revolutionize the field of cancer research and therapy. The ability to precisely target and edit …

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we
investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or …

Gene-editing technology is an emerging therapeutic modality for manipulating the
eukaryotic genome by using target-sequence-specific engineered nucleases. Because of …

Bacteriophages are pervasive viruses that infect bacteria, relying on their genetic machinery
to replicate. In order to protect themselves from this kind of invader, bacteria developed an …

CRISPR/Cas9 system exploits the concerted action of Cas9 nuclease and programmable
single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …

Sickle cell disease (SCD) is an inherited monogenic disorder resulting in serious mortality
and morbidity worldwide. Although the disease was characterized more than a century ago …

Abstract Human induced Pluripotent Stem Cells (iPSCs) have enormous potential in
understanding developmental biology, disease modeling, drug discovery, and regenerative …

Sickle cell disease (SCD) is the most common monogenic hematologic disorder and is
essentially congenital hemolytic anemia caused by an inherited point mutation in the β …

Sickle cell disease (SCD) is one of the most common hereditary hemoglobinopathies
worldwide, affecting almost 400,000 newborns globally each year. It is characterized by …