RNA‐based therapeutics have the potential to revolutionize the treatment and prevention of
human diseases. While early research faced setbacks, it established the basis for …

Sickle cell disease (SCD) is a hereditary blood disorder characterized by the presence of
abnormal hemoglobin molecules and thus distortion (sickling) of the red blood cells. SCD …

Engineered virus-like particles (eVLPs) are promising vehicles for transient delivery of
proteins and RNAs, including gene editing agents. We report a system for the laboratory …

X-linked chronic granulomatous disease (X-CGD) is an inborn error of immunity (IEI)
resulting from genetic mutations in the cytochrome b-245 beta chain (CYBB) gene. 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 …

This article provides an overview of conventional, new, and future treatment options for
sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current …

Therapeutic genome editing of haematopoietic stem cells (HSCs) would provide long-lasting
treatments for multiple diseases. However, the in vivo delivery of genetic medicines to HSCs …

Gene therapy has shown significant potential in treating various diseases, particularly
inherited blood disorders such as hemophilia, sickle cell disease, and thalassemia …

In humans, specific aberrations in β-globin results in sickle cell disease and β-thalassemia,
symptoms of which can be ameliorated by increased expression of fetal globin (HbF). Two …

The rapid advancement of sequencing technologies has led to the identification of
numerous mutations in cancer genomes, many of which are variants of unknown …