CRISPR-Cas technology has revolutionized biological research and holds great therapeutic
potential. Here, we review CRISPR-Cas systems and their latest developments with an …

The CRISPR-Cas9 technology has changed the landscape of genome editing and has
demonstrated extraordinary potential for treating otherwise incurable diseases. Engineering …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …

Nogo receptor 1 is the high affinity receptor for the potent myelin-associated inhibitory
factors that make up part of the inflammatory extracellular milieu during experimental …

Localized immunomodulation technologies are rapidly emerging as a new modality with the
potential to revolutionize transplantation of cells and organs. In the past decade, cell-based …

Programmable nucleases have enabled rapid and accessible genome engineering in
eukaryotic cells and living organisms. However, their delivery into human blood cells can be …

Over the last decade, incrementally improved xenograft mouse models, which support the
engraftment and development of a human hemato-lymphoid system, have been developed …

Due to pioneering in vitro investigations on gene modification, gene engineering platforms
have incredibly improved to a safer and more powerful tool for the treatment of multiple …

Metachromatic leukodystrophy (MLD) is a rare genetic disorder caused by mutations in the
Arylsulfatase-A (ARSA) gene. The enzyme plays a key role in sulfatide metabolism in brain …

Utrophin upregulation is considered a promising therapeutic strategy for Duchenne
muscular dystrophy (DMD). A number of microRNAs (miRNAs) post-transcriptionally …