Thalassaemia is a diverse group of genetic disorders with a worldwide distribution affecting
globin chain synthesis. The pathogenesis of thalassaemia lies in the unbalanced globin …

BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is
now commonplace in academic laboratories and industry for both research and clinical …

The past several years have seen tremendous advances in the engineering of immune
effector cells as therapy for cancer. While chimeric antigen receptors (CARs) have been …

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an
effective therapy for monogenic diseases of the blood and immune system. Here we …

Gene therapies are gaining momentum as promising early successes in clinical studies
accumulate and examples of regulatory approval for licensing increase. Investigators are …

The achievements of cell-based therapeutics have galvanized efforts to bring cell therapies
to the market. To address the demands of the clinical and eventual commercial-scale …

Adipose stem cells (ASCs) have attracted considerable attention as potential therapeutic
agents due to their ability to promote tissue regeneration. However, their limited tissue repair …

Epigenetic mechanisms are known to define cell-type identity and function. Hence,
reprogramming of one cell type into another essentially requires a rewiring of the underlying …

Abstract Recently, natural killer (NK)-based immunotherapy has attracted attention as a next-
generation cell-based cancer treatment strategy due to its mild side effects and excellent …