Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …

Inborn errors of immunity (IEIs) are a group of inherited disorders caused by mutations in the
protein-coding genes involved in innate and/or adaptive immunity. Hematopoietic stem cell …

Abstract Homology-directed repair (HDR), a method for repair of DNA double-stranded
breaks can be leveraged for the precise introduction of mutations supplied by synthetic DNA …

The applicability of nuclease-based form of prime editor (PEn) has been hindered by its
complexed editing outcomes. A chemical inhibitor against DNA-PK, which mediates the …

For precise genome editing via CRISPR/homology-directed repair (HDR), effective and safe
editing of long-term engrafting hematopoietic stem cells (LT-HSCs) is required. The impact …

Introduction Ex vivo gene therapy for treatment of Inborn errors of Immunity (IEIs) have
demonstrated significant clinical benefit in multiple Phase I/II clinical trials. Current …

Genome engineering via targeted nucleases, specifically CRISPR-Cas9, has revolutionized
the field of gene therapy research, providing a potential treatment for diseases of the blood …

As HSC GT is becoming available for more diseases, novel developments should focus on
improving availability while reducing costs of the treatment. Continued follow up of treated …

It is widely understood that cancer is a significant cause of morbidity and mortality
worldwide. Despite numerous available treatments, prognosis for many remains poor, thus …

Lessons learned from decades-long practice in the transplantation of hematopoietic stem
and progenitor cells (HSPCs) to treat severe inherited disorders or cancer, have set the …