The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

There have been many ups and downs since the introduction of gene therapy as a
therapeutic modality for diseases. However, the journey of gene therapy has reached a …

Lipid nanoparticles have attracted significant interests in the last two decades, and have
achieved tremendous clinical success since the first clinical approval of Doxil in 1995. At the …

A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …

Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells
to exert the sustained production of therapeutic proteins or to correct erroneous genes of the …

Molecular, cellular, and clinical studies of human inborn errors of immunity have
revolutionized our understanding of their pathogenesis, considerably broadened their …

Gene therapy has historically been defined as the addition of new genes to human cells.
However, the recent advent of genome-editing technologies has enabled a new paradigm in …

In the past century, the recombinant DNA technology was just an imagination that desirable
characteristics can be improved in the living bodies by controlling the expressions of target …

Background Gene delivery systems are essentially necessary for the gene therapy of human
genetic diseases. Gene therapy is the unique way that is able to use the adjustable gene to …